Vertex Pharmaceuticals jolted Wall Street with a fresh batch of midstage data on one of its combo therapies against cystic fibrosis.
Vertex Pharmaceuticals ($VRTX) kept hopes alive for treating cystic fibrosis patients with the most common genetic mutation behind the lung disease, with new data from a mid-stage study showing improved lung function in patients on a combination of its approved drug Kalydeco and the experimental compound VX-809.
Vertex Pharmaceuticals ($VRTX) has hit an unexpected snag with its cystic fibrosis pill Kalydeco. The FDA is raising questions about a potential risk of cataracts in children using the drug, and asked the drugmaker to conduct a two-year study to assess that risk.
Regulators gave their final blessing to Vertex Pharmaceuticals' cystic fibrosis treatment Kalydeco, Almirall's lung drug Eklira Genuair, and Eisai's Fycompa seizure drug. And they granted Novartis dispensation to market its cancer drug Afinitor for breast cancer.
Vertex Pharmaceuticals has rolled to European approval of its pioneering cystic fibrosis drug Kalydeco as expected, following the early nod for treatment in January from the FDA to treat a subset of patients with the G551D mutation.
About 6 months after the FDA gave the nod to Vertex Pharmaceuticals' pricey drug Kalydeco, the European Commission also approved the treatment for patients with cystic fibrosis that have a certain genetic mutation.
Vertex ($VRTX) today opened the door on a key part of final results for a mid-stage combo study of its two cystic fibrosis drugs, mapping out its late-stage strategy on a program that has built up hopes for a potentially blockbuster pioneering treatment.
Vertex shares took a nasty tumble this morning, freefalling 18% on the news that the company was forced to revise the data on its closely watched cystic fibrosis combo therapy after learning that the initial interim data report had been bungled.
This year the agency and U.S. lawmakers appear to be supportive of a plan that would help pharma groups race to the market with breakthrough drugs.
The data indicate that Vertex might have a remedy for treating a large number of the 70,000 people worldwide with CF.