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.)

Read More

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.

Read More

FDA advisory panel approves novel cholesterol-lowering drug

An advisory committee for the Food and Drug Administration has recommended the approval of a novel, injectable cholesterol-lowering drug called alirocumab, though many committee members have noted certain restrictions for its use and have requested further data on the drug’s ability to reduce the risk of heart problems.

According to the Centers for Disease Control and Prevention (CDC), around 73.5 million people in the US have high levels of “bad” cholesterol, known as low-density lipoprotein (LDL). What is more, less than half of adults with high LDL cholesterol are receiving treatment for the condition.High cholesterol is a major risk factor for heart disease, putting individuals at twice the risk for the condition. Lowering LDL cholesterol can significantly reduce the risk of heart disease, as well as the risk of heart attack, stroke and other cardiovascular conditions.

At present, a class of drugs called statins are the standard treatment for high LDL cholesterol and have been for the past 2 decades. Statins lower levels of LDL cholesterol by interfering with an enzyme called HMG-CoA reductase, which is responsible for cholesterol production in the liver.However, though statins are an effective class of cholesterol-lowering drugs, they can produce a number of side effects, including muscle pain, liver damage, digestive problems and increased blood sugar. Research has also linked statin use to memory loss, though a recent study claims this is not due to the drugs themselves.

Alirocumab (brand name Praluent) lowers LDL cholesterol by inhibiting PCSK9 (proprotein convertase subtilisin/kexin type 9) – a protein that impairs the liver’s ability to remove cholesterol from the blood.According to the drug manufacturers – Regeneron and Sanofi – alirocumab is safer and more effective than statins for reducing high LDL cholesterol.Their conclusion is based on the findings of a phase 3 clinical program involving more than 5,000 patients with high LDL cholesterol, including some who are not able to tolerate statins. The findings revealed a 75-mg dose of Praluent reduces LDL cholesterol by around 50%, while a 150-mg dose results in a 60-65% reduction.

PANELISTS VOTE 13-3 IN FAVOR OF ALIROCUMAB APPROVAL, BUT WITH RESTRICTIONS

In a meeting on Tuesday, the Food and Drug Administration (FDA) advisory committee took the results of the phase 3 clinical program into account when deciding whether to recommend approval for alirocumab as a treatment for patients with high LDL cholesterol.

The committee voted 13-3 in favor of alirocumab approval, claiming that the research has demonstrated that “alirocumab is well-tolerated and provides substantial reductions in LDL-C [LDL cholesterol] for a population of patients whose LDL-C is not adequately controlled with existing therapies.”

However, while Regeneron and Sanofi seek approval for alirocumab as a treatment for a large proportion of patients with high cholesterol, if the drug is given the green light by the FDA, its use may be much more restricted.Some members of the advisory committee said – based on current data – they recommend it be used to treat patients with abnormally high cholesterol triggered by a genetic disorder known as familial hypercholesterolemia (FH), and they said more data is needed on its effectiveness to reduced heart conditions before they can recommend its use in wider populations.Nine of the 16 committee members said they believe alirocumab should be offered to patients at high risk of cardiovascular disease as a result of factors that cannot be controlled with statins.Only seven panelists recommended alirocumab use for patients who are unable to tolerate statins due to side effects, with many of these stating the drug should be used in combination with a tolerable level of statins.

But despite these caveats, Regeneron and Sanofi are pleased with the outcome of the FDA advisory committee’s meeting. Dr. George D. Yancopoulos, chief scientific officer of Regeneron and president of Regeneron Laboratories, says:

“The discovery of PCSK9 as a powerful regulator of cholesterol levels and cardiovascular disease was one of the most important human genetic advances of the last decade.

Today’s outcome brings us one step closer to translating this genetics-based discovery into a treatment that may help the many patients in need of additional cholesterol lowering.”

It is estimated that if the FDA does grant approval for alirocumab, the drug would cost around $7,000-$12,000 per patient annually, while brand-name statins cost around $500-$7,000 per patient each year.Talking to CNN, however, Dr. Eliiott Antman, president of the American Heart Association, said alirocumab can be a cost-effective treatment option for patients with high cholesterol.”If we [balance] the cost of these drugs over society and the cost to our health care system for caring for patients who suffer the consequences of vascular disease,” he said, “it’s possible that the calculus would suggest this could be a cost effective and attractive approach.”

The FDA will make their final decision on whether to approve alirocumab later this summer, though they are expected to follow the recommendation of the advisory committee.

Today, the FDA advisory committee is meeting to make recommendations on another PCSK9 inhibitor called evolocumab (brand name Repatha) – manufactured by Amgen Inc.

Read More

Alectinib approved by FDA for ALK-positive lung cancer

Alectinib, a new drug for anaplastic lymphoma kinase (ALK)-positive non-small cell lung cancer, has been approved by the US Food and Drug Administration (FDA).Alectinib, marketed as Alecensa by Genentech, has been licensed for patients who cannot tolerate crizotinib or whose disease has worsened after treatment with the drug.Alectinib was studied in two trials involving patients with metastatic disease for whom crizotinib, marketed as Xalkori by Pfizer, was no longer effective.

In the first study, 38% of patients had partial shrinkage of their tumours, lasting 7.5 months. In the second study, 44% of patients had partial tumour shrinkage lasting 11.2 months on average. For most patients, alectinib was effective at shrinking metastases in the brain, one of the most common places for lung cancer to spread.About 4-5% of non-small cell lung cancer patients are ALK-positive — the gene mutation is usually found in younger patients with no or light smoking history. The ALK subtype is the result of a chromosomal rearrangement that creates a novel gene that drives the formation of cancer.

Crizotinib, the first drug to target ALK-positive lung cancer, was approved by the FDA in November 2013 and by the European Medicines Agency (EMA) in November 2012. Since the approval of crizotinib, ceritinib has also been approved in the United States and Europe, making alectinib the third drug to be licensed for the disease.“[This] approval provides a new therapy for a group of patients who would have few treatment options once their disease no longer responds to treatment with Xalkori,” says Richard Pazdur, director of the Office of Hematology and Oncology Products at the FDA.Alectinib is currently under evaluation by the EMA.The main side effects associated with alectinib are fatigue, constipation, swelling and muscle pain.

Read More

FDA approves Treatment for: Irritable Bowel Syndrome with Diarrhea (IBS-D)

Viberzi

Generic Name: eluxadoline
Date of Approval: May 27, 2015
Company: Actavis plc

Treatment for: Irritable Bowel Syndrome with Diarrhea (IBS-D)

Viberzi is a first in class treatment for irritable bowel syndrome with diarrhea, treating the characteristic symptoms of abdominal pain and diarrhea.

The U.S. Food and Drug Administration (FDA) has approved Viberzi (eluxadoline), a twice-daily, oral treatment for adults suffering from irritable bowel syndrome with diarrhea (IBS-D). Viberzi has mixed opioid receptor activity (mu receptor agonist, delta receptor antagonist, and kappa receptor agonist), and the FDA has recommended classification as a controlled substance.

MEDICATION GUIDE

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

IMPORTANT INFORMATION

Viberzi can cause serious side effects, including:

• Sphincter of Oddi spasm. The sphincter of Oddi is a muscular valve that controls the flow of digestive juices (bile and pancreatic juice) to the first part of your small intestine. A sphincter of Oddi spasm can cause an increase in your liver and pancreas enzymes and inflammation of the pancreas (pancreatitis) that can cause sudden stomach-area (abdomen) pain. Your risk of having a sphincter of Oddi spasm is increased if you do not have a gallbladder. This spasm usually happens within the first week of treatment with Viberzi and usually goes away when treatment is stopped.
  Stop taking Viberzi and call your doctor if you have new or worsening stomach-area (abdomen) pain or pain in the upper right side of your stomach-area (abdomen) that may move to your back or shoulder, with or without nausea and vomiting.
• Inflammation of the pancreas (pancreatitis). Symptoms of pancreatitis usually go away when treatment with Viberzi is stopped. Your risk of getting pancreatitis is increased if you drink more than three alcoholic drinks a day. Limit your use of alcoholic drinks during treatment.
  Stop taking Viberzi and call your doctor if you have new or worsening stomach-area (abdomen) pain that may move to your back or shoulder, with or without nausea and vomiting.

WHAT IS VIBERZI?

Viberzi is a prescription medicine used to treat adults who have irritable bowel syndrome with diarrhea (IBS-D).

Viberzi is a controlled substance (CX) because it contains eluxadoline that can be a target for people who abuse prescription medicines or street drugs. Keep your prescription in a safe place, to protect it from theft. Never give your Viberzi to anyone else, because it may harm them. Selling or giving away this medicine is against the law.

It is not known if this medicine is safe and effective in children.

An increased number of side effects, including serious side effects and stomach problems, have been reported in people 65 years and older.

WHO SHOULD NOT TAKE VIBERZI?

Do not take this medicine if you:

• have or may have had a blockage in your gallbladder or a sphincter of Oddi problem
• have or had problems with alcohol abuse, alcohol addiction, or drink more than three alcoholic drinks a day
• have had inflammation of your pancreas (pancreatitis) or other pancreas problems, including if you have had or may have had a blockage in your pancreas
• have severe liver problems
• have had long-lasting (chronic) or severe constipation, or problems caused by constipation
• have or may have had a bowel blockage (intestinal obstruction)

Talk to your doctor if you are not sure if you have any of these conditions.

BEFORE TAKING VIBERZI

Before taking this medicine, tell your doctor about all of your medical conditions, including if you:

• See Important information
• do not have a gallbladder
• have liver problems
• are pregnant or plan to become pregnant. It is not known if this medicine will harm your unborn baby.
• are breastfeeding or plan to breastfeed. It is not known if this medicine passes into your breast milk or could harm your baby.

Tell your doctor about all the medicines you take, including prescription and over-the-counter medicines, vitamins, and herbal supplements. Keep a list of your medicines to show your doctor and pharmacist when you get a new medicine. Viberzi and other medicines may affect each other causing side effects.

If you are taking Viberzi you should not take:

• medicines that cause constipation including:
  • Lotronex (alosetron)
  • anticholinergic medicines
  • opioid pain medicines
  Ask your doctor or pharmacist for a list of these medicines, if you are not sure.
• Avoid taking loperamide, a medicine used to treat diarrhea, for a long time (chronic use). You may take loperamide occasionally to treat severe diarrhea. Stop taking loperamide right away if you become constipated.

HOW SHOULD I TAKE VIBERZI?

• Take Viberzi exactly as your doctor tells you to take it.
• Take ONE tablet, two times each day with food.
• If you miss a dose, take your next dose at your regular time. Do not take two doses at the same time to make up for a missed dose.
• Do not change your dose or stop treatment unless your doctor tells you to.
• If you overdose, call your doctor or go to the nearest hospital emergency room right away.

WHAT SHOULD I AVOID WHILE TAKING VIBERZI?

• Limit your use of alcoholic drinks while you are taking Viberzi.
• If you have liver problems, do not drive, operate machinery, or do other dangerous activities until you know how Viberzi affects you.

VIBERZI SIDE EFFECTS

See Important information

The most common side effects include: constipation, nausea, and abdominal pain. Stop treatment and call your doctor if you have constipation that lasts more than four days.

These are not all the possible side effects. 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 VIBERZI?

Store tablets at room temperature between 68°F to 77°F (20°C to 25°C).

Keep all medicines out of the reach of children.

GENERAL INFORMATION ABOUT THE SAFE AND EFFECTIVE USE OF VIBERZI

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 to other people, even if they have the same symptoms that you have. It may harm them. You can ask your doctor or pharmacist for information that is written for health professionals.

WHAT ARE THE INGREDIENTS IN VIBERZI?

Active ingredient: eluxadoline

Inactive ingredients: silicified microcrystalline cellulose, colloidal silica, crospovidone, mannitol, magnesium stearate, and Opadry II (partially hydrolyzed polyvinyl alcohol, titanium dioxide, polyethylene glycol, talc, iron oxide yellow, and iron oxide red).

Read More

FDA Approves Orkambi (lumacaftor/ivacaftor) for Cystic Fibrosis

ORKAMBI

Generic Name: ivacaftor and lumacaftor
Date of Approval: July 2, 2015
Company: Vertex Pharmaceuticals Incorporated

Treatment for: Cystic Fibrosis

July 2, 2015 — The U.S. Food and Drug Administration today approved the first drug for cystic fibrosis directed at treating the cause of the disease in people who have two copies of a specific mutation.

Orkambi (lumacaftor 200 mg/ivacaftor 125 mg) is now approved to treat cystic fibrosis (CF) in patients 12 years and older, who have the F508del mutation, which causes the production of an abnormal protein that disrupts how water and chloride are transported in the body. Having two copies of this mutation (one inherited from each parent) is the leading cause of CF.

“The FDA encourages manufacturers to develop new and innovative treatments for serious rare diseases like cystic fibrosis,” said John Jenkins, M.D., director of the Office of New Drugs, Center for Drug Evaluation and Research. “Today’s approval significantly broadens the availability of targeted treatments for the specific defects that cause cystic fibrosis.”

Orkambi received FDA’s breakthrough therapy designation because the sponsor demonstrated through preliminary clinical evidence that the drug may offer a substantial improvement over available therapies. The FDA also reviewed Orkambi under the priority review program. A priority review is conducted over six months, or less, instead of the standard 10 months, and is employed for drugs that may offer significant improvement in safety or effectiveness in treatment over available therapy in a serious disease or condition.

In addition, the FDA granted Orkambi orphan drug designation because it treats cystic fibrosis, a rare disease. Orphan drug designation provides financial incentives, like clinical trial tax credits, user fee waivers, and eligibility for market exclusivity to promote rare disease drug development.

CF is a serious genetic disorder that results in the formation of thick mucus that builds up in the lungs, digestive tract and other parts of the body leading to severe respiratory and digestive problems, as well as other complications such as infections and diabetes.

CF, which affects about 30,000 people in the United States, is the most common fatal genetic disease in Caucasians. The F508del mutation is the most common cause of CF. People who have two copies of the F508del mutation, one inherited from each parent, account for approximately half of the CF population in the U.S.

The safety and efficacy of Orkambi was studied in two double-blind, placebo-controlled clinical trials of 1,108 participants with CF who were 12 years and older with the F508del mutation. In both studies, participants with CF who took Orkambi, two pills taken every 12 hours, demonstrated improved lung function compared to those who took placebo.

The efficacy and safety of Orkambi have not been established in patients with CF other than those with the F508del mutation. If a patient’s genotype is unknown, an FDA cleared CF mutation test should be used to detect the presence of the F508del mutation on both alleles of the CFTR gene.

The most common side effects of Orkambi include shortness of breath, upper respiratory tract infection, nausea, diarrhea, and rash. Women who took Orkambi also had increased menstrual abnormalities such as increased bleeding.

PATIENT INFORMATION

Read this Patient Information before you start treatment and each time you get a refill. There may be new information. This information does not take the place of talking with your doctor about your medical condition or your treatment.

WHAT IS ORKAMBI?

Orkambi is a prescription medicine used for the treatment of cystic fibrosis (CF) in patients age 12 years and older who have two copies of the F508del mutation (F508del/F508del) in their CFTR gene. It should not be used in patients other than those who have two copies of theF508del mutation in their CFTR gene.It is not known if this medicine is safe and effective in children under 12 years of age.

WHO SHOULD NOT TAKE ORKAMBI?

Do not take Orkambi if you take certain medicines or herbal supplements such as:

• antibiotics: rifampin (Rifamate, Rifater) or rifabutin (Mycobutin)
• seizure medications: phenobarbital, carbamazepine (Tegretol, Carbatrol, and Equetro), or phenytoin (Dilantin, Phenytek)
• sedatives/anxiolytics: triazolam (Halcion) or midazolam (Dormicum, Hypnovel, and Versed)
• immunosuppressant medicines: everolimus (Zortress), sirolimus (Rapamune), or tacrolimus (Astagraf XL, Envarsus XR, Prograf, Protopic)
• St. John’s wort (Hypericum perforatum)

Talk to your doctor before you start treatment if you take any of the medicines or supplements listed above.

WHAT SHOULD I TELL MY DOCTOR?

Before you start treatment, tell your doctor if you:

• have or have had liver problems
• have kidney problems
• are using birth control (hormonal contraceptives, including oral, injectable, transdermal, or implantable forms). Hormonal contraceptives should not be used as a method of birth control when taking Orkambi. Talk to your doctor about the best birth-control method you should use.
• are pregnant or plan to become pregnant. It is not known if this medicine will harm your unborn baby. You and your doctor should decide if you will take Orkambi while you are pregnant.
• are breastfeeding or planning to breastfeed. It is not known if this medicine passes into your breast milk. You and your doctor should decide if you will take Orkambi while you are breastfeeding.

Orkambi may affect the way other medicines work, and vice versa. Tell your doctor about all the medicines you take, including prescription and over-the-counter medicines, vitamins, and herbal supplements, because the dose of Orkambi may need to be adjusted when taken with certain medications.

Ask your doctor or pharmacist for a list of these medicines if you are not sure.

Especially tell your doctor if you take:

• antifungal medications such as ketoconazole (Nizoral), itraconazole (Sporanox), posaconazole (Noxafil), or voriconazole (Vfend)
• antibiotics such as telithromycin (Ketek), clarithromycin (Biaxin), or erythromycin (Ery-Tab)

Know the medicines you take. Keep a list of them to show your doctor and pharmacist when you get a new medicine.

HOW SHOULD I TAKE ORKAMBI?

• Take Orkambi exactly as your doctor tells you to take it.
• Always the tablets with fat-containing foods such as eggs, avocados, nuts, butter, peanut butter, cheese pizza, whole-milk dairy products, (such as whole milk, cheese, and yogurt), etc.
• Take your doses twelve hours apart.
• Each box contains four weekly cartons. Each carton contains seven daily blister strips. Each blister strip contains four tablets so you can take two tablets for the morning and two tablets for the evening.
• You may cut along the dotted line to separate your morning dose from your evening dose.
• Unpeel the paper backing from a blister strip to remove two tablets and take them with fat-containing food in the morning, and repeat again in the evening, twelve hours later.
• If you miss a dose within six hours of when you usually take it, take your dose with fat-containing food as soon as possible.
• If you miss a dose and it is more than six hours after the time you usually take it, skip that dose only and take the next dose when you usually take it. Do not take two doses at the same time to make up for your missed dose.
• Tell your doctor if you stop treatment for more than one week. Your doctor may need to change your dose of Orkambi or other medicines you take.

WHAT SHOULD I AVOID WHILE TAKING ORKAMBI?

It is unknown if Orkambi causes dizziness. Do not drive a car, use machinery, or do anything that needs you to be alert until you know how it affects you.

ORKAMBI SIDE EFFECTS

Orkambi can cause serious side effects.

High liver enzymes in the blood, which can be a sign of liver injury, have been reported in patients receiving Orkambi.

Your doctor will do blood tests to check your liver:

• before you start treatment
• every three months during your first year of treatment
• every year while you are taking Orkambi

Call your doctor right away if you have any of the following symptoms of liver problems:

• pain or discomfort in the upper right stomach (abdominal) area
• yellowing of your skin or the white part of your eyes
• loss of appetite
• nausea or vomiting
• dark, amber-colored urine
• confusion

Respiratory events such as shortness of breath or chest tightness were observed in patients when starting Orkambi. If you have poor lung function your doctor may monitor you more closely when you start treatment.

Abnormality of the eye lens (cataract) has been noted in some children and adolescents receiving ivacaftor, a component of Orkambi. Your doctor should perform eye examinations prior to and during treatment to look for cataracts.

The most common side effects include:

• shortness of breath and/or chest tightness
• upper respiratory tract infection (common cold), including sore throat, stuffy or runny nose
• gastrointestinal symptoms, including nausea, diarrhea, or gas
• rash
• fatigue
• flu or flu-like symptoms
• increase in muscle enzyme levels
• irregular, missed, or abnormal periods (menses) and increase in the amount of menstrual bleeding

Tell your doctor if you have any side effect that bothers you or that does not go away.

These are not all the possible side effects. For more information, ask your doctor or pharmacist.

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 ORKAMBI?

• Store Orkambi at room temperature between 68°F to 77°F (20°C to 25°C).

Do not use Orkambi after the expiration date on the package.

Keep all medicines out of the reach of children.

GENERAL INFORMATION ABOUT THE SAFE AND EFFECTIVE USE OF ORKAMBI.

Medicines are sometimes prescribed for purposes other than those listed in a Patient Information leaflet. Do not use this medicine for a condition for which it was not prescribed. Do not give it to other people, even if they have the same symptoms you have. It may harm them.

This Patient Information summarizes only the most important information. If you would like more information, talk with your doctor. You can ask your pharmacist or doctor for information that is written for health professionals.

WHAT ARE THE INGREDIENTS IN ORKAMBI?

Active ingredients: lumacaftor and ivacaftor

Inactive ingredients: cellulose, microcrystalline; croscarmellose sodium; hypromellose acetate succinate; magnesium stearate; povidone; and sodium lauryl sulfate.

The tablet film coat contains: carmine, FD&C Blue #1, FD&C Blue #2, polyethylene glycol, polyvinyl alcohol, talc, and titanium dioxide.

The printing ink contains: ammonium hydroxide, iron oxide black, propylene glycol, and shellac.

Read More