– For the first time, up to 10,000 people in Europe ages 12 years and older with one F508del mutation and one minimal function mutation will be eligible for a medicine that treats the underlying cause of cystic fibrosis – – People 12 years of age and older who have two F508del mutations will also
- More than 600 people with certain rare CF mutations could become newly eligible for TRIKAFTA, SYMDEKO or KALYDECO - BOSTON --(BUSINESS WIRE)--Sep. 1, 2020-- Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced the U.S. Food and Drug Administration (FDA) accepted three supplemental
LONDON --(BUSINESS WIRE)--Sep. 10, 2020-- The Vertex Foundation , a nonprofit charitable foundation, today announced a €1 million donation to Ronald McDonald House Charities (RMHC) to support the construction of a Ronald McDonald House at the new children’s hospital in Dublin, Ireland .
BOSTON --(BUSINESS WIRE)--Sep. 10, 2020-- Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced the company has completed a global Phase 3 study of TRIKAFTA ® (elexacaftor/tezacaftor/ivacaftor and ivacaftor) in children ages 6 through 11 years old with cystic fibrosis (CF) who have
LONDON --(BUSINESS WIRE)--Sep. 14, 2020-- Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced the European Medicines Agency (EMA) has validated a Type II Variation Marketing Authorization Application (MAA) for the expanded indication of KAFTRIO ®* (ivacaftor/tezacaftor/elexacaftor)
Second collaboration between Moderna and Vertex based on Moderna’s proprietary mRNA and LNP technology Collaboration will leverage Vertex’s investments and capabilities in genetic technologies for CF Moderna to receive $75 million upfront, with potential for additional development, regulatory and
ZUG, Switzerland and CAMBRIDGE, Mass. and BOSTON, Sept. 22, 2020 (GLOBE NEWSWIRE) -- CRISPR Therapeutics (Nasdaq: CRSP) and Vertex Pharmaceuticals Inco r porated (Nasdaq: VRTX) today announced the European Medicines Agency (EMA) has granted Priority Medicines (PRIME) designation to CTX001, an
- Oral presentation of interim results from TRIKAFTA ® (elexacaftor/tezacaftor/ivacaftor and ivacaftor) open-label safety extension study to be presented at the ECFS Digital Conference - - Six presentations highlighting data from KALYDECO ® (ivacaftor), ORKAMBI ® (lumacaftor/ivacaftor) and TRIKAFTA
-Approval provides opportunity to treat the underlying cause of CF earlier than ever before- -Safety data from a cohort of the Phase 3 ARRIVAL study support treatment with KALYDECO in children ages four to