An independent advisory committee to the U.S. FDA voted in favor of approving Vertex Pharmaceuticals’ (VRTX) new treatment for Cystic Fibrosis, Orkambi, on Tuesday afternoon. The agency will make an official approval decision by July 5.
VRTX was halted during Tuesday’s trading session, but healthcare investors sent sector-tracking ETFs higher into the closing bell. At $30 billion, VRTX makes up about 4% of the iShares NASDAQ Biotech ETF (IBB).
The FDA’s Pulmonary-Allergy Advisory Committee voted 12 to 1 in support of approving Orkambi, a combination of lumacaftor and ivacaftor, for the treatment of cystic fibrosis patients with the F508del mutation. The panel voted 13 to 0 that the product is safe, but on the question “Do the data show that lumacaftor contributes positively to the clinical efficacy of Orkambi?” the panel voted 3-Yes, 4-No, and 6-Can’t determine.
This was, perhaps, the overarching theme going into the panel meeting – does lumacaftor’s addition to the approved ivacaftor (sold as Kalydeco) really improve outcomes vs ivacaftor alone in F508del patient?
Kalydeco (ivacaftor) is approved for a small portion of the CF population globally, those with the G551D or RH117 mutations. Orkambi combines Kalydeco with the corrector lumacaftor to treat patients with the most common form of CF, those that are F508del homozygous. About half of the 70,000 people with CF globally have this form of the disease.
The question now – how much can Vertex charge? Kalydeco already costs about $300K annually.
