Category: Therapies

Source: Cystic Fibrosis Foundation

Vertex Pharmaceuticals Inc. today announced promising interim results from a Phase 2 clinical trial of its cystic fibrosis drug Kalydeco™ and VX-809, a CF drug in development.

The results showed a significant improvement in lung function in people with two copies of the most common CF mutation who received the two drugs in combination.

Both Kalydeco and VX-809 are designed to treat the underlying cause of CF. Complete results from the Phase 2 trial are expected this summer.

The ongoing Phase 2 study enrolled 108 people, ages 18 and older, who have one or two copies of the Delta F508 mutation. Today’s results are based on data from about half of the study participants after they had completed 56 days of treatment.

Vertex plans to begin a pivotal trial of Kalydeco and VX-809 in people with two copies of the Delta F508 mutation, pending final study results. Pivotal trials are typically designed to gather data that could be used by the U.S. Food and Drug Administration (FDA) to decide whether or not to approve a potential drug.

“We are eagerly awaiting the full results, and are pleased that Vertex is accelerating its plans for a pivotal study of the combination treatment in those with two copies of Delta F508,” said Robert J. Beall, Ph.D., president and CEO of the CF Foundation.

About 50 percent of people with CF in the United States have two copies of the Delta F508 mutation. About 40 percent of people with CF in the United States have one copy.

Earlier this year, the FDA approved Kalydeco for people with the G551D mutation ages 6 and older. Kalydeco is the first drug that treats the underlying cause of CF — a defective gene and its protein product, known as CFTR.

Digestive Care, Inc. (DCI), announced it has received U.S. Food and Drug Administration (FDA) approval of its new drug application for PERTZYE™, indicated for the treatment of exocrine pancreatic insufficiency due to cystic fibrosis or other conditions.

PERTZYE is a unique pancreatic enzyme product containing bicarbonate-buffered enteric-coated microspheres and is protected by several U.S. and international patents. The PERTZYE formulation was previously marketed by DCI for over a decade under the trade name PANCRECARB® MS-16.

Dr. Tibor Sipos, President and Chief Scientific Officer at DCI, stated, “The approval of PERTZYE represents a significant milestone for DCI. This achievement confirms our commitment to the continued development of products vital to the wellbeing of patients living with chronic diseases.”

The short-term safety and efficacy of PERTZYE were evaluated in a randomized, multicenter, double-blind, placebo-controlled, crossover study conducted in patients ages 8 to 43 years with EPI due to CF.1 The primary efficacy endpoint was the mean difference in coefficient of fat absorption (CFA) between PERTZYE and placebo treatment. Mean CFA was 83% with PERTZYE treatment compared to 46% with placebo treatment (p<0.001).

“The improvement in mean CFA observed in the controlled study represents a clinically meaningful treatment benefit for patients using PERTZYE. Availability of this unique buffered formulation of pancreatic enzyme is an important addition to the therapeutic options for CF and other patients with EPI,” stated Michael W. Konstan, MD, Chairman of Pediatrics, Rainbow Babies and Children’s Hospital and Case Western Reserve University School of Medicine, Cleveland, Ohio.

Additional information on the approval of PERTZYE can be found in the FDA Updated Questions and Answers for Healthcare Professionals and the Public: Use an Approved Pancreatic Enzyme Product (PEP) at http://www.fda.gov/Drugs/DrugSafety/PostmarketDrugSafetyInformationforPatientsandProviders/ucm204745.htm.

Source: FDA news release

Two new pancreatic enzyme products used to help aid food digestion, Ultresa (pancrelipase) and Viokace (pancrelipase), were approved March 1 by the U.S. Food and Drug Administration.

Ultresa is a delayed-release capsule used to treat children and adults with cystic fibrosis, a serious genetic disorder affecting the lungs and other organs, or other conditions who cannot digest food normally because their pancreas does not make enough pancreatic enzymes.

Viokace, in combination with a proton pump inhibitor, is used to treat adults who cannot digest food normally. Adults with chronic pancreatitis, a continuing, chronic inflammatory process of the pancreas, or those who have had some or all of their pancreases removed (pancreatectomy) may not digest food normally because they lack needed enzymes or because their enzymes are not released into the bowel (intestine). Viokace’s safety and efficacy in children has not been established.

“The approvals of Ultresa and Viokace, along with the other approved pancreatic enzyme products, allow health care providers to prescribe the product that is most appropriate for the estimated 200,000 patients in the United States who have pancreatic insufficiency,” said Julie Beitz, M.D., director of the Office of Drug Evaluation III in FDA’s Center for Drug Evaluation and Research.

Ultresa and Viokace are the fourth and fifth pancreatic enzyme products approved by FDA. Other FDA-approved pancreatic enzyme products include Creon (2009), Zenpep (2009) and Pancreaze (2010). Approved pancreatic enzyme products meet FDA standards for safety, efficacy and product quality.

Unapproved pancreatic enzyme products had been available for many years. FDA established a date of April 28, 2010 for the makers of pancreatic enzyme products to stop manufacturing and distributing unapproved products.

Ultresa and Viokace are marketed by Bridgewater, N.J.-based Aptalis Pharma U.S. Inc.

Source: Gilead website

February 15, 2012

 

Important Information Regarding the Availability of Cayston®

To the CF Community,

Gilead Sciences, Inc., the manufacturer of Cayston, is facing significant and unanticipated challenges manufacturing enough Cayston to meet demand in the United States.

Gilead is committed to patient care. To that end, we have consulted with physicians who specialize in the treatment of cystic fibrosis and members of the patient advocacy community to make them aware of the shortage and to get their input on our process for managing this situation.

Patients who are currently active on Cayston therapy are our first priority, and every effort is being made to avoid any interruptions in their therapy. This group of patients includes only those who have received 2 or more courses of Cayston in the last 6 months or 1 or more courses in the last 2 months. For all other patients, pharmacies will continue to accept prescriptions for Cayston, but they will not be filled until Gilead can ensure that there is sufficient supply. Patients and their providers should discuss alternative treatment options to Cayston while this supply shortage is in effect. For an individual patient who has no feasible alternative to Cayston, their healthcare provider may request an exception. These cases will be considered on a case-by-case basis through a separate medical review process.

Gilead takes this issue very seriously, and we are working urgently to resolve the supply shortage. However, at this time, we do not know how long the shortage will last.

Guidance for Patients

• Patients who are currently taking Cayston should complete their course of Cayston as prescribed by their healthcare providers.
• Patients who have received 2 or more courses of Cayston in the last 6 months or 1 or more courses in the last 2 months will be able to continue receiving refills unless there are further changes in our supply.
• Patients should call their specialty pharmacy when their next refill of Cayston is due. Pharmacies will continue to fill prescriptions in accordance with when a patient is due to initiate their next course of treatment.
• Patients who do not meet the requirements as defined above should discuss alternative treatment options with their providers.
• If an individual patient has a significant clinical need for Cayston with no feasible alternatives, their healthcare provider can request an exception for that patient. This will be considered through a separate medical review process and communicated back to the patient’s healthcare provider.
• Once the supply issue is resolved, we will inform the CF community about timing for Cayston availability so that patients and their healthcare providers can determine the next steps in their care.
• Cayston will not be available in hospitals.

Treatment alternatives

Our goal is to maintain the supply of Cayston to patients currently active on Cayston therapy, but we cannot guarantee it. As such, Gilead recommends that patients and their healthcare providers consider alternative treatment options until the Cayston shortage is resolved. Please be aware that there are no equivalent forms of aztreonam for inhalation solution approved by the FDA that can be used in place of Cayston.

Actions we are taking

At Gilead, the welfare of patients is our highest priority. We are very conscious of the potential impact of this shortage on people living with CF. We are actively working to qualify new and expand existing manufacturing sites to increase the production of Cayston. We are committed to keeping the CF community informed of our progress as updates become available.

Updates and inquiries about the Cayston drug supply

To stay up-to-date about the availability of Cayston:

• Register for e-mail updates at www.Cayston.com/supply.html
• Call the Cayston Access Program at 1-877-7CAYSTON between the hours of 8AM-8PM ET

Thank you for your support as we work through this issue.

Sincerely,

Noreen R. Henig, MD
Sr. Director, Medical Affairs
Respiratory and PAH Therapeutics
Gilead Sciences, Inc.

Source: PMD Healthcare press release

PMD Healthcare Inc., announced today the launch and availability of its novel new lung health monitoring device – Spiro PD. Recently cleared by the U.S. Food and Drug Administration (FDA), Spiro PD is the first and only personal spirometer that enables patients with lung diseases – those with asthma, chronic obstructive pulmonary disease (COPD), cystic fibrosis (CF) and lung transplants – to easily and accurately monitor their lung function any time and anywhere.

As the first and only personal spirometer, the easy-to-use device is designed specifically to monitor lung function of an adult, adolescent or young child. Other features of Spiro PD allow patients to view their lung function trends over time, manage medications, set reminder alarms to take medicine, do breathing exercises and quickly upload data to their computer and share it with their doctor.

“With the increase in prevalence of emergency room visits and hospitalizations for asthma and other pulmonary problems in the U.S., the availability of Spiro PD is especially significant,” explained Dr. Michael S. Blaiss, a member of the Board of Directors of the World Allergy Organization and a Clinical Professor of Pediatrics and Medicine, University of Tennessee Health Sciences Center in Memphis. “This new device allows patients and parents of young children with lung diseases to know exactly how their condition is doing at any time, enabling patients to have a more active role in controlling their lung health, and potentially identifying problems before the need for costly emergency treatment.”

Lung, or pulmonary, disease is any disease or disorder that occurs in the lungs or that causes the lungs to not work properly¹. Regular measurement and monitoring of lung function is important to pulmonary disease management.

“We are excited about the launch of Spiro PD, as we were able to utilize the latest electronic technology to provide patients with lung diseases with an easy-to-use device that conveniently allows them to monitor their lung function anytime and anywhere,” said Wayne Meng, Founder, Chief Executive Officer and President of PMD Healthcare, Inc. “We are confident that this innovative, portable and affordable personal spirometer will empower patients to better control their disease, by enhancing medication adherence, improving communication between doctor and patient and avoiding expensive ER trips and hospital stays.”

About the Spiro PD

The Spiro PD (“Spiro” stands for spirometer, a device used to measure the volume and flow of air entering and leaving the lungs² and “PD” stands for personal device), is the first personal spirometer that enables patients with lung diseases – those with asthma, COPD, CF and lung transplants – to easily and accurately monitor their lung function anytime and anywhere. Spiro PD allows patients to view their lung function trends over time, manage medications, set alarms reminding them to take medicine, do breathing exercises and quickly upload data to their computer and share it with their doctor.

Spiro PD is cleared to market by the FDA for the use by a patient to test lung function in children, adolescents and adults. It is a single-patient device. Spiro PD is also certified with the CE mark for the European Union (EU) market. Spiro PD meets American Thoracic Society (ATS) and European Respiratory Society (ERS) standards.

Spiro PD is available online with a prescription. Spiro PD is designed and marketed by PMD Healthcare, Inc. For more information visit www.spiropd.com.

About Lung Disease

Lung, or pulmonary, disease is any disease or disorder that occurs in the lungs or that causes the lungs to not work properly1. One example is COPD, the number three cause of death in the U.S. according to the Centers for Disease Control and Prevention (CDC)³. Regular measurement and monitoring of lung function is important to pulmonary disease management, including:

• Asthma is a chronic lung disease that inflames and narrows the airways and can be a life-threatening illness if not properly managed⁴. An estimated 25 million Americans, including nearly 7 million children, are currently living with asthma⁵. Annually, asthma accounts for approximately 17 million doctor office visits, including physician offices, hospital outpatient and emergency departments⁶, 10 million missed work days and 13 million missed school days⁷.
• Chronic Obstructive Pulmonary Disease (COPD), which includes chronic bronchitis and emphysema, is a progressive lung disease that obstructs the airway or damages the small air sacs in the lungs. These changes restrict airflow into and out of the lungs and result in breathing difficulty. More than 12 million Americans are estimated to have COPD, and an estimated additional 12 million adults are undiagnosed.
• Cystic fibrosis (CF) is a fatal, inherited chronic disease that causes severe lung damage and nutritional deficiencies. Approximately 30,000 children and adults in the U.S. are living with this disease and more than 10 million Americans are carriers of the CF gene. About 1,000 new cases of CF are diagnosed each year⁸.
• Lung transplant involves a surgical procedure in which a patient’s diseased lungs are partially or totally replaced by lungs from a donor. It is usually used as a last resort for lung failure⁹.

About PMD Healthcare, Inc.

PMD Healthcare, Inc., headquartered in Allentown, Pennsylvania, is dedicated to creating innovative, easy-to-use, portable and affordable personal medical devices, and to empower people worldwide to improve their healthcare and quality of life. For more information about PMD Healthcare, Inc. visit www.personalmedicaldevices.com.

References

1. American Lung Association. Lung Disease. http://www.lungusa.org/lung-disease/ Acces.sed January 2012.
2. American Lung Association. Tools for Identifying & Diagnosing Patients at Risk for COPD. http://www.lungusa.org/associations/states/iowa/events-programs/ia-copd-coalition/ia-copd-assets/tools-for-identifying.pdf Acces.sed January 2012.
3. American Lung Association. Understanding COPD. http://www.lungusa.org/lung-disease/copd/about-copd/understanding-copd.html Acces.sed January 2012.
4. American Lung Association. Understanding Asthma. http://www.lungusa.org/lung-disease/asthma/about-asthma/understanding-asthma.html Acces.sed January 2012.
5. National Heart Lung and Blood Institute. What is Asthma?. http://www.nhlbi.nih.gov/health/prof/lung/asthma/naci/asthma-info/index.htm Acces.sed January 2012.
6. Centers for Disease Control and Prevention. Asthma: FastStats. http://www.cdc.gov/nchs/fastats/asthma.htm Acces.sed January 2012.
7. National Heart Lung and Blood Institute. National Asthma Control Initiative. http://www.nhlbi.nih.gov/health/prof/lung/asthma/naci/pubs/naci-factsheet.pdf Acces.sed January 2012.
8. American Lung Association. Understanding Cystic Fibrosis. http://www.lungusa.org/lung-disease/cystic-fibrosis/understanding-cystic-fibrosis.html Acces.sed January 2012.
9. National Heart Lung and Blood Institute. What is a Lung Transplant?. http://www.nhlbi.nih.gov/health/health-topics/topics/lungtxp/ Acces.sed January 2012.

Source: Vertex Pharmaceuticals press release

Vertex Pharmaceuticals Incorporated announced today that the U.S. Food and Drug Administration (FDA) has approved KALYDECO™ (ivacaftor), the first medicine to treat the underlying cause of cystic fibrosis (CF), a rare, genetic disease.

KALYDECO (kuh-LYE-deh-koh) is approved for people with CF ages 6 and older who have at least one copy of the G551D mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene. Approximately 1,200 people in the United States, or 4 percent of those with CF, are believed to have this mutation. KALYDECO was granted approval in approximately three months, making it one of the fastest FDA approvals ever and marking the second approval of a new medicine from Vertex in less than a year. The company has established a financial assistance and patient support program to help get KALYDECO to eligible patients for whom it is prescribed. KALYDECO was discovered as part of a collaboration with Cystic Fibrosis Foundation Therapeutics, Inc., the nonprofit drug discovery and development affiliate of the Cystic Fibrosis Foundation.

Vertex is ready to support the introduction of KALYDECO and will begin shipping it to pharmacies in the United States this week.

“More than 13 years ago we set out to change the lives of people with cystic fibrosis by developing new medicines that address the underlying cause of this rare and devastating disease,” said Jeffrey Leiden, M.D., Ph.D., Vertex’s incoming President and Chief Executive Officer. “KALYDECO represents a major advance in the treatment of cystic fibrosis for people with a specific type of this disease. But our work isn’t done. With the ongoing support of doctors, patients and the Cystic Fibrosis Foundation, we’re making progress toward our ultimate goal of developing additional medicines to help many more people with cystic fibrosis.”

The approval of KALYDECO was based on data from two Phase 3 studies of people with CF who have at least one copy of the G551D mutation. Those who were treated with KALYDECO experienced significant and sustained improvements in lung function as well as other disease measures, including weight gain and certain quality of life measurements, compared to those who received placebo. People who took KALYDECO also experienced significantly fewer pulmonary exacerbations, which are periods of worsening in the signs and symptoms of the disease that often require treatment with antibiotics and hospital visits. Fewer people in the KALYDECO treatment groups discontinued treatment due to adverse events than in the placebo groups. The majority of adverse events associated with KALYDECO were mild to moderate. Adverse events commonly observed in those taking KALYDECO included headache, upper respiratory tract infection (common cold), stomach pain and diarrhea.

“Advances in cystic fibrosis treatment have helped manage symptoms of the disease, however people with cystic fibrosis still have a hard time staying healthy and being active,” said Bonnie Ramsey, M.D., Director of the Center for Clinical and Translational Research at Seattle Children’s Research Institute and principal investigator for one of the Phase 3 KALYDECO trials. “KALYDECO is a fundamental shift in the way cystic fibrosis is treated. In people with a specific genetic mutation, KALYDECO helped them breathe more easily, gain weight and generally feel better.”

“Together, we’re changing the lives of people with cystic fibrosis,” said Robert J. Beall, Ph.D., President and CEO of the Cystic Fibrosis Foundation. “We now have a medicine that treats the underlying cause of the disease in people with the G551D mutation. KALYDECO also provides us with a roadmap for exploring additional targeted approaches to treatment for all people with cystic fibrosis.”

Cystic fibrosis is a rare, life-threatening genetic disease for which there is no cure. CF is caused by defective or missing CFTR proteins resulting from mutations in the CFTR gene. CFTR proteins act as channels at the cell surface that control the flow of salt and water across the cells. When the defective CFTR protein does not work properly at the cell surface, abnormally thick, sticky mucus builds up in the lungs. The digestive tract and a number of other organs are also affected. KALYDECO, an oral medicine known as a CFTR potentiator, helps the CFTR protein function more normally once it reaches the cell surface. KALYDECO targets the abnormal CFTR protein channels and opens them to allow chloride ions to move into and out of the cell, which helps thin the mucus so it can hydrate and protect the airways, and keeps them from getting clogged and then infected.

Because KALYDECO targets a specific genetic mutation, a person’s genotype should be known before this new medicine is prescribed. Genetic testing is widely available and FDA-cleared tests are available for people with CF whose genotype is unknown. According to the 2010 Cystic Fibrosis Foundation’s Patient Registry, nearly 92 percent of people with CF have already had their CF mutations identified.

KALYDECO by itself works in a subset of people with CF, but research is ongoing to explore a similar targeted approach using a combination of medicines, including KALYDECO, to treat the most common form of the disease.

Helping People with CF Get KALYDECO

The people who work at Vertex understand that medicines can only help patients who can get them. To that end, the company offers a comprehensive financial assistance and patient support program. A specially-trained and dedicated Vertex team will provide one-on-one support to help eligible patients who are prescribed KALYDECO understand their insurance benefits and the resources that are available to help them.

For eligible patients, the program also includes the following:

Free Medicine Program: Vertex will provide KALYDECO for free to people who do not have insurance and have an annual household income of $150,000 or less; and Co-Pay Assistance Program: For patients with commercial insurance plans that cover KALYDECO and who are enrolled in the Guidance and Patient Support, or GPS, program, there will be a minimal out-of-pocket obligation after which Vertex will help cover co-pay or co-insurance costs up to 30 percent of the list price of the medicine. There is no income limit to be eligible for this program. Some patients are not eligible for company co-pay support because they have Medicare or Medicaid coverage or live in Massachusetts. There are independent non-profit copay assistance foundations that may be able to help those patients with their out-of-pocket costs.

More information about this program is available by calling 1-877-7-KALYDECO (877-752-5933) or visiting www.VertexGPS.com.

About KALYDECO

KALYDECO is the first treatment to target the underlying cause of CF. The Phase 3 studies evaluated KALYDECO in people with CF ages 6 and older who had at least one copy of the G551D mutation. PERSIST, a Phase 3, open-label, 96-week extension study, is underway to evaluate the long-term safety and durability of treatment with KALYDECO. This ongoing study enrolled people who completed 48 weeks of treatment in either Phase 3 study (placebo and KALYDECO treatment groups) and met other eligibility criteria. KALYDECO will be taken as one 150-mg tablet twice daily (every 12 hours).

Vertex retains worldwide rights to develop and commercialize KALYDECO. In October 2011, Vertex submitted a marketing authorization application to the European Medicines Agency (EMA) for KALYDECO and has received agreement from the EMA for accelerated assessment in Europe. The EMA regulatory review is ongoing.

Indication and Important Safety Information

KALYDECO is a prescription medicine used for the treatment of cystic fibrosis (CF) in patients ages 6 years and older who have a certain mutation in their CF gene called the G551D mutation.

KALYDECO is not for use in people with CF due to other mutations in the CF gene. It is not effective in CF patients with two copies of the F508del mutation (F508del/F508del) in the CF gene.

It is not known if KALYDECO is safe and effective in children under 6 years of age.

KALYDECO should not be used with certain medicines, including the antibiotics rifampin and rifabutin; seizure medications (phenobarbital, carbamazepine, or phenytoin); and the herbal supplement St. John’s Wort.

KALYDECO can cause serious side effects. High liver enzymes in the blood have occurred in patients taking KALYDECO. Regular assessment is recommended.

The most common side effects associated with KALYDECO include headache; upper respiratory tract infection (common cold) including sore throat, nasal or sinus congestion, and runny nose; stomach (abdominal) pain; diarrhea; rash; nausea; and dizziness.

These are not all the possible side effects of KALYDECO. Patients should tell their healthcare providers about any side effect that bothers them or doesn’t go away.

Please see full Prescribing Information for KALYDECO at www.KALYDECO.com.

Source: Cystic Fibrosis Foundation

An expanded access program for a potential new CF drug, Kalydeco™ (VX-770), is now available at participating clinical sites throughout the country for people with the G551D mutation who have highly limited lung function and may benefit from treatment.

Vertex Pharmaceuticals, Inc., the maker of Kalydeco (kuh-LYE-deh-koh), created this program for patients while awaiting review of the drug by the U.S. Food and Drug Administration (FDA).

The company is seeking FDA approval for Kalydeco in people ages 6 and older with at least one copy of the G551D mutation.

People with CF who may be eligible for this program and want more information should talk to their CF doctor or call the Vertex Expanded Access Call Center at 1-800-745-4484.

Kalydeco is an oral drug in development that targets the underlying cause of cystic fibrosis. Kalydeco was developed by Vertex with CF Foundation support and research input.

Souce: Vertex Pharmaceuticals press release

Vertex Pharmaceuticals says it plans to begin additional studies of KALYDECO™ in children with cystic fibrosis as young as two years of age and in people with CF who have certain mutations that were not evaluated in the previous Phase 3 studies. Pending final feedback from regulatory agencies, the company plans to begin three clinical studies of KALYDECO in mid-2012.

In December, Vertex announced that the U.S. Food and Drug Administration (FDA) accepted the New Drug Application for KALYDECO and granted the company’s request for six-month Priority Review. A target review date of April 18, 2012, is set under the Prescription Drug User Fee Act for the FDA’s approval decision. Vertex’s marketing authorization application for KALYDECO has also been validated by the European Medicines Agency, which accepted Vertex’s request for accelerated assessment in Europe.

As Vertex prepares for the potential launch of KALYDECO for people with the G551D mutation, the company is also planning to begin additional studies of KALYDECO in children with CF as young as two years of age and in people with CF who have certain mutations that were not evaluated in the previous Phase 3 studies.

Pending final feedback from regulatory agencies, the company plans to begin three clinical studies of KALYDECO in mid-2012:

• Pediatric study: A study of KALYDECO in children ages 2 through 5 with gating mutations, including G551D, is expected to evaluate the safety, tolerability and effect on sweat chloride and other measures of clinical activity using a pediatric formulation of KALYDECO.

• Study in people with the R117H mutation: Vertex plans to begin the first clinical study of KALYDECO in people who have at least one copy of the R117H mutation in the CF gene. The R117H mutation causes abnormal function of the CFTR protein at the cell surface. Approximately 3 percent of people with CF in the U.S. have the R117H mutation.

• Study in other gating mutations: Vertex also plans to begin the first clinical study of KALYDECO in other gating mutations where CFTR proteins are present at the cell surface but do not function properly. G551D is the most common gating mutation, present in approximately 4 percent of people with CF in the U.S., and was the focus of previous Phase 3 KALYDECO studies. The remaining gating mutations to be evaluated in this study account for an additional approximately 1 percent of people with CF in the U.S.

Additional studies of KALYDECO and Vertex’s pipeline medicines in development are ongoing or planned for 2012, including:

• Two CFTR correctors in development for people with the most common CF mutation, F508del: Enrollment is ongoing in the second part of a Phase 2 clinical trial of combination regimens of KALYDECO, a CFTR potentiator, and VX-809, a CFTR corrector, in people with the most common mutation in CF, known as F508del. In addition, Vertex plans to begin Phase 2 development of VX-661, a second CFTR corrector, in the first quarter of 2012. Data from the study with VX-809 is expected mid-year, followed by data from the study with VX-661 later in 2012.