Novartis $2 million gene therapy for rare disorder is world's most expensive drug

Swiss drugmaker Novartis on Friday won U.S. approval for its gene therapy Zolgensma for spinal muscular atrophy (SMA), the leading genetic cause of death in infants, and priced the one-time treatment at a record $2.125 million.The Food and Drug Administration approved Zolgensma for children under the age of two with SMA, including those not yet showing symptoms. The approval covers babies with the deadliest form of the inherited disease as well as those with types where debilitating symptoms may set in later.“This is potentially a new standard of care for babies with the most serious form of SMA,” said Dr. Emmanuelle Tiongson, a pediatric neurologist at Children’s Hospital Los Angeles who has provided Zolgensma to patients under an expanded access program. “The job now is trying to negotiate with insurers that this would be a long-term savings.”Novartis executives defended the price, saying that a one-time treatment is more valuable than expensive long-term treatments that cost several hundred thousand dollars a year.

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FDA Approves Krintafel (tafenoquine) for the Radical Cure of Plasmodium vivax Malaria

 GSK and Medicines for Malaria Venture (MMV) today announced that the United States Food and Drug Administration (FDA) has approved, under Priority Review, single-dose Krintafel (tafenoquine) for the radical cure (prevention of relapse) of Plasmodium vivax (P. vivax) malaria in patients aged 16 years and older who are receiving appropriate antimalarial therapy for acute P. vivax infection.Dr. Hal Barron, Chief Scientific Officer and President of Research and Development, GSK, said: “Today’s approval of Krintafel, the first new treatment for Plasmodium vivax malaria in over 60 years, is a significant milestone for people living with this type of relapsing malaria. Together with our partner, Medicines for Malaria Venture, we believe Krintafel will be an important medicine for patients with malaria and contribute to the ongoing effort to eradicate this disease.”

Dr. David Reddy, Chief Executive Officer of MMV said: “The US FDA’s approval of Krintafel is a major milestone and a significant contribution towards global efforts to eradicate malaria. The world has waited decades for a new medicine to counter P. vivax malaria relapse. Today, we can say the wait is over. Moreover, as the first ever single-dose for this indication, Krintafel will help improve patient compliance. We are proud to have worked side-by-side with GSK for more than a decade to reach this point. Our focus is now on working to ensure the medicine reaches the vulnerable patients that need it most.”

The approval was based on efficacy and safety data from a comprehensive global clinical development P. vivax radical cure programme designed in agreement with the FDA. Thirteen studies in healthy volunteers and patients directly supported the programme. The primary evidence for the clinical efficacy and safety of the 300mg single-dose, to which more than 800 subjects were exposed, was provided by three randomised, double-blind studies: DETECTIVE Part 1 and Part 2 (TAF112582) and GATHER (TAF116564). The results of the two phase III studies were announced in June 2017. The submission included data analysed from a total of thirty-three studies involving more than 4,000 trial subjects exposed to the 300mg single-dose and other doses of tafenoquine.With the approval of Krintafel, the FDA awarded GSK a tropical disease priority review voucher. The tropical disease priority review voucher programme is designed to encourage development of new drugs and biological products for prevention and treatment of certain neglected tropical diseases affecting millions of people throughout the world.The new drug application (NDA) was submitted by GSK to the FDA in November 2017 and a regulatory submission was also made to the Australian Therapeutics Good Administration (TGA) in December 2017. A decision from the TGA is awaited. Approvals by FDA and TGA will be informative to other regulatory agencies for their own approval process in malaria-endemic countries where tafenoquine will be provided as a not-for-profit medicine to maximise access to those who need it most.

About Krintafel (tafenoquine)

Krintafel is an 8-aminoquinoline derivative with activity against all stages of the P. vivax lifecycle, including hypnozoites. It was first synthesised by scientists at the Walter Reed Army Institute of Research in 1978. GSK’s legacy in the research and development of tafenoquine as a potential medicine for malaria commenced over 20 years ago. In 2008, GSK entered into a collaboration with the not-for-profit drug research partnership, MMV, to develop tafenoquine as an anti-relapse medicine for patients infected with P. vivax. The tafenoquine clinical programme is part of GSK’s global health programme aimed at improving healthcare for vulnerable populations.

Important Safety Information

CONTRAINDICATIONS

Krintafel should not be administered to:

• patients who have glucose-6-phosphate dehydrogenase (G6PD) deficiency or have not been tested for G6PD deficiency
• patients who are breastfeeding a child known to have G6PD deficiency or one that has not been tested for G6PD deficiency
• patients who are allergic to tafenoquine or any of the ingredients in Krintafel or who have had an allergic reaction to similar medicines containing 8-aminoquinolines.

WARNINGS AND PRECAUTIONS

Hemolytic Anemia Breakdown of red blood cells (hemolytic anemia) may occur in a patient who has G6PD deficiency. G6PD testing must be performed before prescribing Krintafel. Treatment with Krintafel is contraindicated in patients with G6PD deficiency or unknown G6PD.

G6PD Deficiency in Pregnancy or Lactation The use of Krintafel during pregnancy may cause hemolytic anemia in a G6PD-deficient fetus. Krintafel is not recommended during pregnancy. Females of reproductive potential should avoid pregnancy or use effective contraception for 3 months after the dose of Krintafel.

A G6PD-deficient infant may be at risk for hemolytic anemia from exposure to Krintafel through breast milk. Infant G6PD status should be checked before breastfeeding begins. Krintafel is contraindicated in breastfeeding women when the infant is found to be G6PD deficient or the G6PD status of the infant is unknown. A woman with a G6PD-deficient infant or if the G6PD status of the infant is not known should not breastfeed for 3 months after the dose of Krintafel.

Methemoglobinemia Asymptomatic elevations in methemoglobin have been observed in the clinical trials of Krintafel. Physicians should carefully monitor individuals with nicotinamide adenine dinucleotide (NADH)-dependent methemoglobin reductase deficiency and advise patients to seek medical attention if signs of methemoglobinemia occur.

Psychiatric Effects Serious psychiatric adverse reactions have been observed in patients with a previous history of psychiatric conditions at doses higher than the approved dose. The benefit of treatment with Krintafel must be weighed against the potential risk for psychiatric adverse reactions in patients with a history of psychiatric illness. Due to the long half-life of Krintafel (15 days), any signs or symptoms of psychiatric adverse reactions that may occur could be delayed in onset and/or duration.

Hypersensitivity Reactions Serious hypersensitivity reactions (e.g., angioedema) have been observed with administration of Krintafel. Due to the long half-life of Krintafel (15 days), any signs or symptoms of hypersensitivity adverse reactions that may occur could be delayed in onset and/or duration. Physicians should advise patients to seek medical attention if signs of hypersensitivity occur.

ADVERSE REACTIONS

The most common adverse reactions (≥5%) observed for Krintafel in clinical trials were dizziness, nausea, vomiting, headache, and decreased hemoglobin.

DRUG INTERACTIONS

Physicians should avoid coadministration of Krintafel with drugs that are substrates of organic cation transporter-2 (OCT2) or multidrug and toxin extrusion (MATE) transporters (for example, dofetilide, metformin).

To report SUSPECTED ADVERSE REACTIONS, contact GlaxoSmithKline at 1-888-825-5249 or FDA at 1-800-FDA-1088 or www.fda.gov/medwatch.

GSK - a science-led global healthcare company with a special purpose: to help people do more, feel better, live longer. For further information please visit www.gsk.com

Medicines for Malaria Venture (MMV) - MMV is a leading product development partnership (PDP) in the field of antimalarial drug research and development. Its mission is to reduce the burden of malaria in disease-endemic countries by discovering, developing and facilitating delivery of new, effective and affordable antimalarial drugs.

Since its foundation in 1999, MMV and partners have built the largest portfolio of antimalarial R&D and access projects ever assembled, and brought forward seven new medicines that are already saving lives. MMV's success is based on its extensive partnership network of around 160 active pharmaceutical, academic and endemic-country partners in more than 55 countries.

MMV's vision is a world in which innovative medicines will cure and protect the vulnerable and under-served populations at risk of malaria, and help to ultimately eradicate this terrible disease.

Cautionary statement regarding forward-looking statements

GSK cautions investors that any forward-looking statements or projections made by GSK, including those made in this announcement, are subject to risks and uncertainties that may cause actual results to differ materially from those projected. Such factors include, but are not limited to, those described under Item 3.D 'Principal risks and uncertainties' in the company's Annual Report on Form 20-F for 2017.

Source: GSK

Posted: July 2018

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FDA Approves Viekira XR

AbbVie Receives U.S. FDA Approval of Once-Daily Viekira XR (dasabuvir, ombitasvir, paritaprevir and ritonavir) for the Treatment of Genotype 1 Chronic Hepatitis C

NORTH CHICAGO, Ill., July 25, 2016 /PRNewswire/ -- AbbVie (NYSE: ABBV), a global biopharmaceutical company, today announced that the U.S. Food and Drug Administration (FDA) has approved a New Drug Application (NDA) for Viekira XR (dasabuvir, ombitasvir, paritaprevir and ritonavir) extended-release tablets. Viekira XR is a once-daily, extended-release co-formulation of the active ingredients in Viekira Pak (ombitasvir, paritaprevir, and ritonavir tablets; dasabuvir tablets) and is for the treatment of patients with chronic genotype 1 (GT1) hepatitis C virus (HCV) infection, including those with compensated cirrhosis (Child-Pugh A). Viekira XR is not for people with decompensated cirrhosis.

Viekira XR is the first co-formulated three direct-acting antiviral (DAA) treatment for adult patients with GT1 HCV. Viekira XR is given once-daily as three oral tablets and must be taken with a meal. It is used without ribavirin (RBV) in GT1b patients and in combination with twice daily RBV in GT1a patients. The approval is supported by Phase 3 clinical trials for Viekira Pak which include data that demonstrated 100 percent sustained virologic response 12 weeks following treatment (SVR12) in GT1b patients with 12 weeks of therapy without ribavirin and 95 percent SVR12 in GT1a patients when used with ribavirin for 12 or 24 weeks of therapy.

"AbbVie's work continues to contribute to the transformation of hepatitis C care through our focus on evolving our current therapies as part of our ongoing commitment to patients," said Rob Scott, M.D., vice president, development and chief medical officer, AbbVie. "The approval of Viekira XR provides a new treatment option for genotype 1 hepatitis C patients in the U.S. with clinical trial data using the components of Viekira XR demonstrating 100 percent cure rates in genotype 1b patients."

There are six major HCV genotypes (GT1-6) and GT1 is the most prevalent form of HCV in the U.S., accounting for approximately 74 percent of all cases.1 Hepatitis C continues to be an important public health issue, with the Centers for Disease Control and Prevention (CDC) estimating that in the U.S. approximately 2.7 million people are chronically infected with HCV.2

The approval of Viekira XR is supported by data from seven Phase 3 clinical trials in more than 2,300 patients who received Viekira Pak with or without RBV for 12 or 24 weeks and two bioavailability studies comparing the formulations.

About Clinical Studies

The components of Viekira XR (administered twice daily with a meal) have been studied in seven Phase 3 clinical trials where 1076 subjects (including 181 with compensated cirrhosis) received the recommended regimen of Viekira +/? RBV for 12 weeks, or for 24 weeks in GT1a patients with compensated cirrhosis. Ninety-five to 100 percent achieved SVR12, which means the hepatitis C virus is not detectable in the blood three months after treatment ends. Cure rates varied by the subtype of hepatitis C and whether or not the person had cirrhosis. Individual results may vary.

USE

Viekira XR (dasabuvir, ombitasvir, paritaprevir, and ritonavir) extended-release tablets/Viekira Pak (ombitasvir, paritaprevir, and ritonavir tablets; dasabuvir tablets) (Viekira) are prescription medicines used with or without ribavirin to treat adults with genotype 1 chronic (lasting a long time) hepatitis C (hep C) virus infection.

Viekira can be used in people who have compensated cirrhosis.

Viekira is not for people with advanced cirrhosis (decompensated). If people have cirrhosis, they should talk to a doctor before taking Viekira.

About Viekira XR

The components of Viekira XR* have been studied in a broad range of genotype 1 (GT1) patients with chronic hepatitis C virus (HCV) infection, ranging from treatment-naïve to difficult to treat patients, such as those with compensated (mild, Child-Pugh A) cirrhosis of the liver, HCV/HIV-1 co-infection, liver transplant recipients with normal hepatic function and mild fibrosis, and those who have failed previous treatment with pegylated interferon (pegIFN) and ribavirin (RBV).

The extended-release co-formulation of these components, Viekira XR, consists of 200 mg of dasabuvir, 8.33 mg of ombitasvir, 50 mg of paritaprevir, and 33.33 mg of ritonavir per tablet, and is dosed three tablets once daily. Viekira XR must be taken with a meal, and tablets should be swallowed whole. People should not drink alcohol within four hours of taking Viekira XR. Viekira XR is contraindicated in patients with moderate to severe hepatic impairment (Child-Pugh B and C) due to risk of potential toxicity. Viekira XR is taken for 12 weeks, except in GT1a patients with cirrhosis and all liver transplant recipients with normal hepatic function and mild fibrosis, who should take it for 24 weeks. Ribavirin should be co-administered in GT1a patients and in all patients who have received a liver transplant.

Paritaprevir was discovered during the ongoing collaboration between AbbVie and Enanta Pharmaceuticals (NASDAQ: ENTA) for HCV protease inhibitors and regimens that include protease inhibitors. Paritaprevir is used in combination with AbbVie's ombitasvir with or without dasabuvir for the treatment of hepatitis C.

*Given as a fixed-dose combination of ombitasvir 25mg (an NS5A inhibitor), paritaprevir 150mg (an NS3/4A protease inhibitor), and ritonavir 100mg (an HIV-1 protease inhibitor), dosed once daily with a meal, and dasabuvir 250mg (a non-nucleoside NS5B palm polymerase inhibitor), dosed twice daily with a meal.

About AbbVie's Patient Assistance Program

AbbVie supports patient assistance programs to help qualified people access their needed AbbVie medication at no cost. In 2015, more than 81,000 U.S. patients received AbbVie's medicines at no cost3. For those who qualify, AbbVie plans to offer a patient assistance program for people taking Viekira XR. Since Viekira Pak's approval in 2014, AbbVie has supported access to medication for those living with chronic HCV and facing financial difficulties.

About AbbVie's HCV Clinical Development Program

AbbVie's HCV clinical development program is intended to advance scientific knowledge and the clinical care of people with chronic HCV infection. AbbVie is investigating a pan-genotypic (genotypes 1-6) regimen and is in Phase 3 of clinical development. For more information on AbbVie Phase 3 HCV studies, visit www.clinicaltrials.gov (NCT02243293).

About HCV

Hepatitis C is inflammation of the liver caused by an infection with the hepatitis C virus. It is transmitted when an infected person's blood enters the bloodstream of an uninfected person. The Centers for Disease Control (CDC) estimates that approximately 2.7 million people have chronic HCV infection in the U.S. There are six major HCV genotypes (GT1-6), with genotype 1 (GT1) as the most prevalent form in the U.S. It is estimated that of people infected with chronic HCV, about 5 to 20 percent will go on to develop cirrhosis over a period of 20–30 years, and with HCV-related liver transplants on the rise, HCV is a critical public health issue. Presently, there is no vaccine for HCV infection.

About AbbVie

AbbVie is a global, research-based biopharmaceutical company formed in 2013 following separation from Abbott Laboratories. The company's mission is to use its expertise, dedicated people and unique approach to innovation to develop and market advanced therapies that address some of the world's most complex and serious diseases. Together with its wholly-owned subsidiary, Pharmacyclics, AbbVie employs more than 28,000 people worldwide and markets medicines in more than 170 countries. For further information on the company and its people, portfolio and commitments, please visit www.abbvie.com. Follow @abbvie on Twitter or view careers on our Facebook or LinkedIn page.

Forward-Looking Statements

Some statements in this news release may be forward-looking statements for purposes of the Private Securities Litigation Reform Act of 1995. The words "believe," "expect," "anticipate," "project" and similar expressions, among others, generally identify forward-looking statements. AbbVie cautions that these forward-looking statements are subject to risks and uncertainties that may cause actual results to differ materially from those indicated in the forward-looking statements. Such risks and uncertainties include, but are not limited to, challenges to intellectual property, competition from other products, difficulties inherent in the research and development process, adverse litigation or government action, and changes to laws and regulations applicable to our industry.

Additional information about the economic, competitive, governmental, technological and other factors that may affect AbbVie's operations is set forth in Item 1A, "Risk Factors," of AbbVie's 2015 Annual Report on Form 10-K, which has been filed with the Securities and Exchange Commission. AbbVie undertakes no obligation to release publicly any revisions to forward-looking statements as a result of subsequent events or developments, except as required by law.

1 Wedemeyer H. Hepatitis C. Chapter 80: In: Feldman M, Friedman LS, Brandt LJ, eds. Sleisenger and Fordtran's Gastrointestinal and Liver Disease. Vol 2. 10th ed. Philadelphia, PA: Saunders Elsevier; 2016.
2 Centers for Disease Control and Prevention (CDC). Hepatitis C FAQs for health professionals. http://www.cdc.gov/hepatitis/hcv/hcvfaq.htm#section1. Accessed June 9, 2016.
3 AbbVie 2016 Impact by the Numbers. http://www.abbvie.com/responsibility/home.html

SOURCE AbbVie

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FDA Approves Adlyxin

Sanofi Receives FDA Approval of Adlyxin (lixisenatide) for Treatment of Adults With Type 2 Diabetes

PARIS, July 27, 2016 /PRNewswire/ -- Sanofi announced today that the U.S. Food and Drug Administration (FDA) approved Adlyxin (lixisenatide), a once-daily mealtime GLP-1 receptor agonist injection indicated as an adjunct to diet and exercise for the treatment of adults with type 2 diabetes.

"The approval of Adlyxin reaffirms our continued commitment to addressing the challenges faced by people living with diabetes when trying to reach and maintain their individual blood glucose (HbA1c) targets," said Peter Guenter, Executive Vice President, Head, Global Diabetes & Cardiovascular Business Unit, Sanofi. "We are pleased with this approval, as it offers us the opportunity to continue helping patients treated with basal insulin who remain uncontrolled."

The approval of Adlyxin was based on FDA review of results from the GetGoal clinical program and findings from the ELIXA trial, which successfully addressed the FDA's request to demonstrate CV safety. The GetGoal clinical program, which included 13 clinical trials involving more than 5,000 adults with type 2 diabetes worldwide, evaluated the safety and efficacy of lixisenatide in adults with type 2 diabetes. All studies of the GetGoal program successfully met the primary efficacy endpoint of HbA1c reduction. The most common adverse events reported for Adlyxin included nausea, hypoglycemia and vomiting.

Adlyxin will be available in a disposable pre-filled pen in a single dose of 20 micrograms. Patients will also receive a disposable pre-filled pen in a single dose of 10 micrograms that they should initiate once daily for 14 days. On Day 15, patients will increase dosage to 20 micrograms once daily.

Adlyxin is approved under the proprietary name, Lyxumia® in more than 60 countries and marketed in over 40. Commercial launches include most EU countries, Japan, Brazil, Mexico and India. Adlyxin was in-licensed from Zealand Pharma A/S (NASDAQ OMX Copenhagen: ZEAL), www.zealandpharma.com.

About Adlyxin

Adlyxin is a once-daily glucagon-like peptide-1 receptor agonist (GLP-1 RA) for the treatment of adult patients with type 2 diabetes mellitus as an adjunct to diet and exercise. GLP-1 is a peptide hormone that is released within minutes after eating a meal. It is known to suppress glucagon secretion from pancreatic alpha cells and stimulate glucose-dependent insulin secretion by pancreatic beta cells. Adlyxin increases glucose-dependent insulin release, decreased glucagon secretion, and slows gastric emptying.

About Sanofi Diabetes & Cardiovascular

Diabetes and cardiovascular disease affect millions of people worldwide, with many managing the complex challenges of both. Building on our portfolio evolution, heritage and expertise, Sanofi has a focused business unit dedicated to delivering innovative, value-based medicines and integrated solutions in these therapeutic areas. We are committed to a collaborative approach that involves strategic alliances with professional and patient associations, research institutions and leaders in healthcare and other industries, with the goal of advancing scientific knowledge, driving the convergence of science and technology, helping to improve outcomes and inspiring an evolution in care.

About Sanofi

Sanofi, a global healthcare leader, discovers, develops and distributes therapeutic solutions focused on patients' needs. Sanofi is organized into five global business units: Diabetes and Cardiovascular, General Medicines and Emerging Markets, Sanofi Genzyme, Sanofi Pasteur and Merial. Sanofi is listed in Paris (EURONEXT: SAN) and in New York (NYSE: SNY).

Sanofi Forward-Looking Statements

This press release contains forward-looking statements as defined in the Private Securities Litigation Reform Act of 1995, as amended. Forward-looking statements are statements that are not historical facts. These statements include projections and estimates and their underlying assumptions, statements regarding plans, objectives, intentions and expectations with respect to future financial results, events, operations, services, product development and potential, and statements regarding future performance. Forward-looking statements are generally identified by the words "expects", "anticipates", "believes", "intends", "estimates", "plans" and similar expressions. Although Sanofi's management believes that the expectations reflected in such forward-looking statements are reasonable, investors are cautioned that forward-looking information and statements are subject to various risks and uncertainties, many of which are difficult to predict and generally beyond the control of Sanofi, that could cause actual results and developments to differ materially from those expressed in, or implied or projected by, the forward-looking information and statements. These risks and uncertainties include among other things, the uncertainties inherent in research and development, future clinical data and analysis, including post marketing, decisions by regulatory authorities, such as the FDA or the EMA, regarding whether and when to approve any drug, device or biological application that may be filed for any such product candidates as well as their decisions regarding labelling and other matters that could affect the availability or commercial potential of such product candidates, the absence of guarantee that the product candidates if approved will be commercially successful, the future approval and commercial success of therapeutic alternatives, the Group's ability to benefit from external growth opportunities, trends in exchange rates and prevailing interest rates, the impact of cost containment initiatives and subsequent changes thereto, the average number of shares outstanding as well as those discussed or identified in the public filings with the SEC and the AMF made by Sanofi, including those listed under "Risk Factors" and "Cautionary Statement Regarding Forward-Looking Statements" in Sanofi's annual report on Form 20-F for the year ended December 31, 2015. Other than as required by applicable law, Sanofi does not undertake any obligation to update or revise any forward-looking information or statements.

SOURCE: Sanofi

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Massachusetts General Hospital launches phase II trial of BCG vaccine to reverse type 1 diabetes

FDA approval of trial testing generic vaccine announced at ADA Scientific Sessions

A phase II clinical trial testing the ability of the generic vaccine bacillus Calmette-Guérin (BCG) to reverse advanced type 1 diabetes has received approval from the U.S. Food and Drug Administration (FDA). The approval of this trial, which will shortly begin enrolling qualified patients, was announced today at the 75th Scientific Sessions of the American Diabetes Association (ADA) by Denise Faustman, MD, PhD, director of the Massachusetts General Hospital (MGH) Immunobiology Laboratory and principal investigator of the study.

The five-year trial will investigate whether repeat BCG vaccination can clinically improve type 1 diabetes in adults between 18 and 60 years of age who have small but still detectable levels of insulin secretion from the pancreas. Faustman’s research team was the first group to document reversal of advanced type 1 diabetes in mice and subsequently completed a successful phase I human clinical trial of BCG vaccination. She announced the FDA approval to launch the phase II trial during her ADA presentation, “Low Levels of C-Peptide Have Clinical Significance for Established Type 1 Diabetes.”

“We have learned a lot since the early studies in mice – not just about how BCG works but also about its potential therapeutic benefits, similar to what are being seen in trials against other autoimmune diseases,” says Faustman. “We are so grateful to all of the donors, large and small, who have made this trial possible – especially the Iacocca Foundation, which has believed in us and has been a supporter since our early days. Our goal is to complete enrollment and also to raise the remaining funds needed for the trial by the end of this year.”

A generic drug with over 90 years of clinical use and safety data, BCG is currently approved by the FDA for vaccination against tuberculosis and for the treatment of bladder cancer. The vaccine is known to elevate levels of the immune modulator tumor necrosis factor (TNF), which Faustman’s team previously showed can temporarily eliminate in both humans and mice the abnormal white blood cells responsible for autoimmune type 1 diabetes. Increased TNF levels also stimulated production of protective regulatory T cells.

In the phase I clinical trial, which was published in the August 8, 2012, issue of PLOS Medicine, two injections of BCG spaced four weeks apart led to temporary elimination of diabetes-causing T cells and provided evidence of a small, transient return of insulin secretion. The phase II clinical study will include more frequent dosing over a longer time period to determine the potential of repeat BCG vaccination to ameliorate the autoimmune state and improve clinical parameters such as HbA1c, a marker of average blood sugar control.

In the new trial, which will be double blinded and conducted at MGH, 150 adults with long-term type 1 diabetes will be randomized to receive two injections four weeks apart of either BCG or placebo and then a single injection annually for the next four years. Patients will be closely monitored over the five-year trial period. The primary outcome measure will be improved results on the HbA1c blood test, which have been shown to prevent complications.

“In the phase I clinical trial we demonstrated a statistically significant response to BCG, but our goal in phase II is to create a lasting therapeutic response,” says Faustman, an associate professor of Medicine at Harvard Medical School. “We will be working again with people who have had type 1 diabetes for many years. This is not a prevention trial; instead, we are trying to create a regimen that will treat even advanced disease. In addition to our phase I trial, we took guidance from the BCG clinical trials that are underway globally for other autoimmune diseases such as multiple sclerosis.”

Lee Iacocca, founder of the Iacocca Foundation, says, “My family and I have been fortunate to be part of this research for many years. It is incredibly exciting to be talking about curing people, not mice. I made a promise to my late wife to find a cure for type 1 diabetes. Now my family and I look forward to the continued progress and are proud to support this effort to get closer to that goal.” The Iacocca Foundation provided major funding for the phase I trial and has taken a leadership role in funding the phase II trial.

More than $19 million has been raised out of a total of $25 million needed to conduct the phase II study over the next five years. Additional information about the clinical trial, including information for potential participants and financial supporters, is available athttp://www.faustmanlab.org/ or by emailing DiabetesTrial@partners.org. Details on the trial and enrollment also are available at: https://clinicaltrials.gov/ct2/show/NCT02081326.

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FDA Approves Antiplatelet Agent Kengreal (cangrelor) as Adjunct to Percutaneous Coronary Intervention

June 22, 2015 — The U.S. Food and Drug Administration today approved Kengreal (cangrelor), an intravenous antiplatelet drug that prevents formation of harmful blood clots in the coronary arteries, the blood vessels that supply blood to the heart. It is approved for adult patients undergoing percutaneous coronary intervention (PCI), a procedure used to open a blocked or narrowed coronary artery to improve blood flow to the heart muscle.

According to the Centers for Disease Control and Prevention, PCI is performed on approximately 500,000 people in the United States each year. The coronary arteries are opened by inflating a balloon at the site of the narrowing, usually followed by placement of a small mesh tube, called a stent, to keep the artery open.By preventing platelets from accumulating, Kengreal reduces the risk of serious clotting complications related to the procedure, including heart attack and clotting of the stent (stent thrombosis).“For patients undergoing percutaneous coronary intervention, blood clotting can cause serious problems,” said Norman Stockbridge, M.D., Ph.D., director of the Division of Cardiovascular and Renal Drugs in the FDA’s Center for Drug Evaluation and Research. “The approval of Kengreal provides another treatment option for patients.”

As with other FDA-approved anti-platelet drugs, bleeding, including life-threatening bleeding, is the most serious risk of Kengreal.In a clinical trial that compared Kengreal to Plavix (clopidogrel) in more than 10,000 participants, Kengreal significantly reduced the occurrence of heart attack, the need for further procedures to open the artery and stent thrombosis. The overall occurrence of serious bleeding was low but more common with Kengreal than with clopidogrel. Approximately one in every 170 Kengreal patients had a serious bleed versus approximately one in every 275 clopidogrel patients.

Kengreal is manufactured by The Medicines Company based in Parsippany, New Jersey.

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FDA Approves Stiolto Respimat

Stiolto Respimat

Generic Name: olodaterol and tiotropium bromide
Date of Approval: May 21, 2015
Company: Boehringer Ingelheim Pharmaceuticals, Inc.

Stiolto Respimat is a combination of the long-acting beta2-adrenergic agonist olodaterol (Striverdi Respimat), and the anticholinergic agent tiotropium (Spiriva Respimat).

The U.S. Food and Drug Administration (FDA) has approved Stiolto Respimat (olodaterol and tiotropium bromide) inhalation spray for the long-term, once-daily maintenance treatment of patients with chronic obstructive pulmonary disease (COPD), including chronic bronchitis and emphysema. It is not indicated for the acute symptoms of COPD, or for asthma, and does not replace the use of a rescue inhaler.

Medication Guide

Read this Medication Guide before you start treatment and each time you get a refill. There may be new information. This Medication Guide does not take the place of talking to your healthcare provider about your medical condition or treatment.

Important information

Stiolto Respimat has been approved for the treatment chronic obstructive pulmonary disease (COPD) only. It is not to be used in the treatment of asthma.

Stiolto Respimat can cause serious side effects, including:

• People with asthma who take long-acting beta2-adrenergic agonist (LABA) medicines, such as olodaterol (one of the medicines in Stiolto Respimat), have an increased risk of death from asthma problems.
• It is not known if LABA medicines, such as olodaterol, increase the risk of death in people with COPD.
• Get emergency medical care if:
  • breathing problems worsen quickly
  • you use your rescue inhaler medicine, but it does not relieve your breathing problems

What is Stiolto Respimat?

Stiolto Respimat contains two ingredients: olodaterol, a long-acting beta2-adrenergic agonist (LABA), and tiotropium, an anticholinergic agent.

It is used long term, two puffs once each day, in controlling symptoms in adults with COPD.

LABA and anticholinergic medicines help the muscles around the airways in your lungs stay relaxed to prevent symptoms, such as wheezing, cough, chest tightness, and shortness of breath.

Stiolto Respimat is not for use to treat sudden symptoms of COPD. Always have a rescue medicine with you to treat sudden symptoms. If you do not have a rescue inhaler, contact your healthcare provider to have one prescribed for you.

It is not known if this medicine is safe and effective in people with asthma. It should not be used in children. It is not known if Stiolto Respimat is safe and effective in children.

Who should not use this medicine?

Do not use Stiolto Respimat if:

• you have asthma.
• you are allergic to tiotropium, ipratropium, olodaterol, or any of the ingredients. See the end of this Medication Guide for a complete list of active and inactive ingredients.

Before using Stiolto Respimat

Tell your healthcare provider about all of your health conditions, including if you:

• have heart problems
• have high blood pressure
• have seizures
• have thyroid problems
• have diabetes
• have eye problems, such as glaucoma. This medicine can make your glaucoma worse.
• have prostate or bladder problems, or problems passing urine. This medicine can make these problems worse.
• have any other medical conditions
• are pregnant or planning to become pregnant. It is not known if this medicine can harm your unborn baby.
• are breastfeeding. It is not known if the active ingredients pass into your breast milk and if they can harm your baby.
• are allergic to Stiolto Respimat or any of its ingredients, any other medicines, or food products.

Tell your healthcare provider about all the medicines you take, including prescription and over-the-counter medicines, eye drops, vitamins, and herbal supplements. Stiolto Respimat and certain other medicines may interact with each other. This may cause serious side effects.

Especially tell your healthcare provider if you take:

• anticholinergics (including ipratropium, aclidinium, umeclidinium or another tiotropium-containing product such as Spiriva Respimat or Spiriva HandiHaler)
• atropine

Know the medicines you take. Keep a list of your medicines with you to show your healthcare provider and pharmacist each time you get a new medicine.

How should I use Stiolto Respimat?

• Read the step-by-step instructions contained in the product package.
• The inhaler has a slow-moving mist that helps you inhale the medicine.
• Use Stiolto Respimat exactly as your healthcare provider tells you to use it.
• Use ONE dose (TWO puffs) of Stiolto Respimat, one time each day, at the same time of the day.
• If you miss a dose, take it as soon as you remember. Do not take more than ONE dose (TWO puffs) in 24 hours.
• Do not spray the inhaler in your eyes.
• Always use the new inhaler that is provided with each new prescription.
• Stiolto Respimat does not relieve sudden symptoms of COPD. Always have a rescue inhaler medicine with you to treat sudden symptoms. If you do not have a rescue inhaler medicine, call your healthcare provider to have one prescribed for you.
• Do not stop using this medicine, or other medicines used to control or treat your COPD unless told to do so by your healthcare provider because your symptoms might get worse. Your healthcare provider will change your medicines as needed.
• Do not use Stiolto Respimat:
  • more often than prescribed for you, or
  • with other medicines that contain LABA or an anticholinergic for any reason. Ask your healthcare provider or pharmacist if any of your other medicines are LABA or anticholinergic medicines.
• Call your healthcare provider or get emergency medical care right away if:
  • your breathing problems worsen during treatment
  • you need to use your rescue medicine more often than usual
  • your rescue inhaler medicine does not work as well for you at relieving your symptoms

Stiolto Respimat side effects

Stiolto Respimat can cause serious side effects, including:

• See Important information
• If your COPD symptoms worsen over time do not increase your dose. Call call your healthcare provider instead.
• sudden shortness of breath that may be life-threatening
• serious allergic reactions including rash, hives, swelling of the face, mouth, and tongue, and breathing problems. Call your healthcare provider or get emergency medical care if you get any symptoms of a serious allergic reaction.
• heart problems including fast or irregular heartbeat, palpitations, chest pain, increased blood pressure
• new or worsening eye problems including acute narrow-angle glaucoma. Symptoms of acute narrow-angle glaucoma include eye pain or discomfort, blurred vision, seeing halos or colored images around lights, and red eyes. Call your healthcare provider right away if you have any of these symptoms. Use caution as some of these eye problems can affect your ability to drive and operate appliances and machinery.
• new or worsening urinary retention. Symptoms of urinary retention may include difficulty urinating, painful urination, urinating frequently, or urinating in a weak stream or drips. Call your healthcare provider right away if you have any of these symptoms.
• low blood potassium (which may cause symptoms of muscle spasm, muscle weakness or abnormal heart rhythm)
• high blood sugar

Common side effects of Stiolto Respimat include runny nose, cough, and back pain.

Tell your healthcare provider about any side effect that bothers you or that does not go away.

This is not a complete list of side effects. Ask your healthcare provider or pharmacist for more information.

Call your doctor for medical advice about side effects. You may report side effects to FDA at 1-800- FDA-1088.

How should I store Stiolto Respimat?

• Store Stiolto Respimat at room temperature between 68°F to 77°F (20°C to 25°C).
• Do not freeze the cartridge or the inhaler.
• Keep all medicines out of the reach of children.

General Information about the safe and effective use of Stiolto Respimat

Medicines are sometimes prescribed for purposes other than those listed in a Medication Guide. Do not use this medicine for a condition for which it was not prescribed. Do not give it toother people, even if they have the same condition. It may harm them.

This Medication Guide summarizes the most important information about Stiolto Respimat. If you would like more information, talk with your healthcare provider. You can ask your healthcare provider or pharmacist for information that was written for healthcare professionals.

For more information, including a video demonstration on how to use Stiolto Respimat, go to www.stiolto.com.

What are the ingredients in Stiolto Respimat?

Active ingredients: olodaterol and tiotropium bromide

Inactive ingredients: water for injection, benzalkonium chloride, edetate disodium, and hydrochloric acid

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FDA Approves Fycompa as Adjunctive Treatment for Primary Generalized Tonic-Clonic Seizures

Fycompa

Generic Name: perampanel (per AM pa nel)
Brand Names: Fycompa

What is Fycompa?

Fycompa (perampanel) is an anticonvulsant used to treat seizures.Fycompa is a prescription medicine that is used with other medications to treat partial-onset seizures with or without secondarily generalized seizures, and to treat primary generalized tonic-clonic (PGTC) seizures, in adults and children who are at least 12 years old.Fycompa may also be used for purposes not listed in this medication guide.

Introduction

Anticonvulsant; a noncompetitive α-amino-3-hydroxy-5-methyl-4-isoxazolepropionic acid (AMPA)-type glutamate receptor antagonist.

Uses for Fycompa

Seizure Disorders

Management (in combination with other anticonvulsants) of partial-onset seizures in patients ≥12 years of age with epilepsy.Management (in combination with other anticonvulsants) of primary generalized tonic-clonic seizures in patients ≥12 years of age with epilepsy.Designated an orphan drug by FDA for treatment of Lennox-Gastaut syndrome; not FDA-labeled for this orphan indication.

Fycompa Dosage and Administration

General

• When discontinuing anticonvulsant therapy, gradual withdrawal is recommended to minimize potential for increased seizure frequency.1 (See Discontinuance of Anticonvulsants under Cautions.)
• Closely monitor for notable changes in behavior that could indicate the emergence or worsening of suicidal thoughts or behavior or depression. (See Suicidality Risk under Cautions.)

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FDA approves brain implant to help reduce Parkinson’s disease and essential tremor symptoms

June 12, 2015 — The U.S. Food and Drug Administration today approved the Brio Neurostimulation System, an implantable deep brain stimulation device to help reduce the symptoms of Parkinson’s disease and essential tremor, a movement disorder that is one of the most common causes of tremors. The Brio Neurostimulation System can help some patients when medication alone may not provide adequate relief from symptoms such as walking difficulties, balance problems, and tremors.

An estimated 50,000 Americans are diagnosed with Parkinson’s disease each year, according to the National Institutes of Health, and about one million Americans have the condition. The neurological disorder typically occurs in people over age 60, when cells in the brain that produce a chemical called dopamine become impaired or die. Dopamine helps transmit signals between the areas of the brain that produce smooth, purposeful movement — like eating, writing and shaving.

Essential tremor affects several million people and usually occurs in those over age 40. “There are no cures for Parkinson’s disease or essential tremor, but finding better ways to manage symptoms is essential for patients,” said William Maisel, M.D., M.P.H., acting director of the Office of Device Evaluation at the FDA’s Center for Devices and Radiological Health. “This new device adds to the array of treatment options to help people living with Parkinson’s and essential tremor enjoy better, more productive lives.”

The Brio Neurostimulation System consists of a small (1.9in x 2.1in x 0.4in) battery-powered, rechargeable electrical pulse generator implanted under the skin of the upper chest and wire leads that attach to electrodes placed within the brain at specific locations depending on whether the device is being used to treat Parkinson’s disease or essential tremor. The electrical pulse generator continuously delivers low intensity electrical pulses to target areas in the brain. Health care providers make adjustments to the pulse generator to optimize the effects of the Brio Neurostimulation System.

Data supporting the safety and effectiveness of the device system included two clinical studies. One study included 136 patients with Parkinson’s disease and the other included 127 patients with essential tremor. In both studies, patients had symptoms, including tremors, that were not adequately controlled with drug therapy.

The Brio Neurostimulation System was used in addition to medication for patients with Parkinson’s disease and the majority of patients with essential tremor who used the device were able to control their symptoms without the need for medications. Researchers implanted the Brio Neurostimulation System in all patients and assessed effectiveness for Parkinson’s disease patients at three months and essential tremor patients at six months. Both groups showed statistically significant improvement on their primary effectiveness endpoint when the device was turned on compared to when it was turned off.

Serious adverse events included intracranial bleeding, which can lead to stroke, paralysis or death. Other device-related adverse events included infection and dislocation of the device lead under the skin. The Brio Neurostimulation System is manufactured by St. Jude Medical in St. Paul, Minnesota.

Brio Neurostimulation System is the second device approved by the FDA for Parkinson’s and essential tremor. The first device, Medtronic’s Activa Deep Brain Stimulation Therapy System, was approved in 1997 for tremor associated with essential tremor and Parkinson’s disease. In 2002, the indications were expanded to include the symptoms of Parkinson’s disease.

In its early stages, Parkinson’s disease typically affects one side of the body and starts as problems with movement, stiffness, and mild tremors. Gradually, the symptoms can affect both sides of the body and medications may become less effective. People with late stage Parkinson’s disease have many symptoms including: trouble walking, impaired posture and balance, muscle stiffness and tremors in the arms and hands that make it difficult to perform everyday tasks.

Essential tremor most often affects the hands and arms and can be slowly progressive, starting on one side of the body but eventually affecting both sides. Hand tremor is the most common symptom, but tremors can also affect movement in the head, arms, voice, tongue, legs, and trunk. About half of essential tremor cases result from a genetic mutation. For the remainder of cases, the cause is unknown.

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