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Vertex Pharmaceuticals, Inc.

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U.S. FDA Accepts for Priority Review Supplemental New Drug Application for the Use of ORKAMBI® (lumacaftor/ivacaftor) in Children with Cystic Fibrosis Ages 6 to 11 who have Two Copies of the F508del Mutation
-Approximately 2,400 children ages 6 to 11 have two copies of the F508del mutation in the U.S.-

-Target review date of September 30, 2016 set for the FDA's decision on the application-

BOSTON--(BUSINESS WIRE)-- Vertex Pharmaceuticals Incorporated (Nasdaq:VRTX) today announced that the U.S. Food and Drug Administration (FDA) has accepted for review a supplemental New Drug Application (sNDA) for the use of ORKAMBI® (lumacaftor/ivacaftor) in people with cystic fibrosis (CF) ages 6 to 11 who have two copies of the F508del mutation. The FDA granted Vertex's request for Priority Review of this sNDA, and a target review date of September 30, 2016 was set under the Prescription Drug User Fee Act (PDUFA).

"As complications related to CF can occur early in life, we believe it is important to begin treatment as early as possible and this supplemental New Drug Application for approval of ORKAMBI in children as young as six is an important step in that direction," said Jeffrey Chodakewitz, M.D., Executive Vice President and Chief Medical Officer at Vertex. "This submission demonstrates Vertex's continued progress toward our goal of developing medicines for all people with CF."

The sNDA is based on data from an open label Phase 3 clinical safety study of ORKAMBI. Data from this study will be presented at the 39th European Cystic Fibrosis Society conference being held June 8 to 11 in Basel, Switzerland.

To support potential approval in the European Union, a six-month Phase 3 efficacy study of children ages 6 to 11 is ongoing. Vertex recently completed enrollment in this study and, pending data from the study, plans to submit a Marketing Authorization Application (MAA) variation in the European Union in the first half of 2017 for children ages 6 to 11 who have two copies of the F508del mutation. The primary endpoint of this efficacy study is the absolute change in lung clearance index (LCI). There are approximately 3,400 children ages 6 to 11 who have two copies of the F508del mutation in the European Union.
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Source: press release, 5/31/16. http://investors.vrtx.com/releasedetail.cfm?ReleaseID=973361

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Phase 3 Data Support Submission of Supplemental New Drug Application for ORKAMBI in Children Ages 6 to 11 with Two Copies of the F508del Mutation in the U.S.

Vertex recently completed a Phase 3 clinical study that was conducted to support the potential approval of ORKAMBI for children with CF as young as six years of age with two copies of the F508del mutation in the U.S. Children in the study received a twice-daily fixed-dose combination of lumacaftor (200mg) and ivacaftor (250mg) for six months. The study enrolled 58 children ages 6 to 11 with two copies of the F508del mutation, and the primary endpoint of the study was safety. The study did not include a placebo control arm. Secondary and exploratory endpoints evaluated the effect on multiple efficacy endpoints including forced expiratory volume in one second (FEV1), lung clearance index and others.

The study met its primary safety endpoint, and safety data from the study showed that the combination was generally well-tolerated. The most common adverse events were cough, headache, infective pulmonary exacerbation, nasal congestion, abdominal pain, increased sputum and elevated liver enzymes. Two patients (3.4%) discontinued treatment because of adverse events.

Based on these data, Vertex plans to submit a supplemental New Drug Application (sNDA) to the U.S. Food and Drug Administration (FDA) in the second quarter of 2016. There are approximately 2,400 children ages 6-11 who have two copies of the F508del mutation in the U.S.

In addition to meeting its primary safety endpoint, the study showed a 2.5 percentage point improvement in FEV1 (p=0.067), a secondary endpoint, and a -0.88 improvement in lung clearance index (p=0.0018), which was an exploratory endpoint. Improvements in secondary endpoints of body mass index, the respiratory domain of the CF questionnaire-revised (CFQ-R) and sweat chloride were also observed in the study.

To support approval in the European Union, a six-month Phase 3 efficacy study is ongoing to evaluate lumacaftor/ivacaftor in approximately 200 children. The primary endpoint of this study is the absolute change in lung clearance index. There are approximately 3,400 children ages 6-11 who have two copies of the F508del mutation in the European Union.
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Source: press release, 1/27/16. http://investors.vrtx.com/releasedetail.cfm?ReleaseID=952025

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Compound/DeviceSpecialtyIndicationCompound ClassTarget
ORKAMBI (Lumacaftor (VX-809) / KALYDECO (Ivacaftor))Medical GeneticsCystic FibrosisCFTR protein potentiatorCFTR protein

Mechanism of action: ORKAMBI (Lumacaftor (VX-809) / KALYDECO (Ivacaftor)), an investigational oral potentiator, is designed to act directly on the malfunctioning CFTR protein to help restore the balance of salt and water. VX-809 is a novel oral CFTR (Cystic Fibrosis Transmembrane Conductance Regulator) corrector designed to increase the concentration of F508del CFTR proteins at the cell surface.

Phase of Development: Filed

Event Type: Regulatory FDA: PDUFA DATE

Dates: 2016-09-30

Results:

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September 28, 2016
U.S. Food and Drug Administration Approves ORKAMBI® (lumacaftor/ivacaftor) for Use in Children with Cystic Fibrosis Ages 6 through 11 who have Two Copies of the F508del Mutation
-Approximately 2,400 children ages 6 through 11 have two copies of the F508del mutation in the U.S.-

- Vertex revises ORKAMBI revenue guidance for 2016 -

BOSTON--(BUSINESS WIRE)-- Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced that the U.S. Food and Drug Administration (FDA) approved ORKAMBI® (lumacaftor/ivacaftor) for use in children with cystic fibrosis (CF) ages 6 through 11 who have two copies of the F508del mutation. People with this mutation represent the largest population of those with CF, a rare, life-threatening disease. ORKAMBI is the first and only medicine to treat the underlying cause of CF for people with this mutation. It was previously approved by the FDA for use in people ages 12 and older with two copies of the F508del mutation. With today's approval, approximately 11,000 people with CF are eligible for treatment with ORKAMBI in the United States. ORKAMBI will be available for eligible children ages 6 through 11 in the United States as soon as possible. Vertex also today lowered its guidance for 2016 ORKAMBI revenues to a range of $950 million to $990 million.

"The ability to treat children as young as six who have the most common form of the disease is an important milestone as we pursue our goal to develop medicines for all people with CF," said Jeffrey Chodakewitz, M.D., Executive Vice President and Chief Medical Officer at Vertex. "We believe it is important to treat the underlying cause of the disease as early as possible in these patients."

The approval is based on data from a previously announced open-label Phase 3 clinical safety study of ORKAMBI presented at the 39th European Cystic Fibrosis Society Conference in June 2016. These data will be presented at the 30th Annual North American Cystic Fibrosis Conference October 27-29 in Orlando, Florida.

Vertex plans to submit a Marketing Authorization Application (MAA) variation in the European Union in the first half of 2017 for children ages 6 through 11 who have two copies of the F508del mutation. This application will be based on data from a Phase 3 efficacy study with a primary endpoint of absolute change in lung clearance index (LCI). These data are expected before the end of 2016.

ORKAMBI Financial Guidance

Vertex today revised its guidance for 2016 ORKAMBI revenues. The company now expects ORKAMBI revenues of $950 million to $990 million. Vertex's prior guidance was for total 2016 ORKAMBI revenues of $1.0 billion to $1.1 billion, and to date Vertex has reported ORKAMBI revenues of $223 million and $245 million for the first and second quarters of 2016, respectively. Vertex expects third quarter ORKAMBI revenues to be between $230 million and $235 million. The revised guidance primarily reflects the following:

The slower than anticipated launch in Germany where fewer than 20 percent of the approximately 2,500 eligible patients have initiated treatment to date;
That we are approaching peak penetration for ORKAMBI in the U.S. where there are approximately 8,500 eligible patients ages 12 and older; and
Slower than expected refills for ORKAMBI during the summer months of July and August.
Today's approval in people ages 6 through 11 will drive growth in the U.S. in the fourth quarter of 2016. Growth for ORKAMBI in 2017 will be driven both by obtaining reimbursement approvals in key European and other countries and by continued growth among eligible patients ages 6 through 11 in the U.S.
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Source: press release, 9/28/16http://investors.vrtx.com/releasedetail.cfm?ReleaseID=991350

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