- Shares in PTC Therapeutics soared higher in early morning trading Friday after the European Medicines Agency recommended renewal of a conditional approval for the drugmaker's Duchenne muscular dystrophy treatment.
- The decision by the Committee for Medicinal Products for Human Use (CHMP) will allow PTC's Translarna (ataluren) to remain on the market in the European Union, a reprieve for the company after U.S. regulators rejected the drug earlier this year.
- Elsewhere, the CHMP recommended approval for Gilead's new hepatitis B treatment, a combination insulin treatment from Sanofi and three new biosimilars. Unlike the Food and Drug Administration, the CHMP issues approval decisions in a group following a monthly meeting.
PTC had originally expected a decision on renewal to come by mid-year, but discussions with the EMA over draft clinical trial protocols delayed the regulator's assessment. After months of setbacks and layoffs, winning a renewal of Translarna's conditional approval in Europe was critical — sales of Translarna account for all of PTC's product revenue.
Yet PTC will be required to conduct a two-part, three-year study comparing Translarna to a placebo in order to further prove a positive benefit-risk balance. Results from this study are expected by the first quarter of 2021, the CHMP said.
At its November meeting, the CHMP also gave a green light to several important new drugs from Gilead and Sanofi. Following CHMP recommendation, a decision on final approval goes to the European Commission, which usually follows the advice of the agency.
Gilead's Vemlidy (tenofovir alafenamide) further expands the California drugmaker's liver franchise, and gives patients with hepatitis B access to the improved, safer form of tenofovir. (Gilead's existing hepatitis B treatment consists of the older tenofovir disoproxil fumarate.)
While Sanofi's combo of lixisenatide and Lantus (insulin glargine) has been delayed in the U.S., the French drugmaker was able to secure the backing of the CHMP for marketing authorization in Europe. Sanofi has been battling rival Novo Nordisk, which has its own combination of a GLP-1 and long-acting insulin.
Both companies are awaiting a decision by the FDA in the U.S., although Novo won approval for its Xultolphy (insulin degludec/liraglutide) in Europe back in 2014.
Sanofi is working to build out its diabetes franchise as top-selling Lantus faces a growing competitive threat from biosimilars. That threat was underscored Friday by the CHMP's recommendation for approval of Merck Sharp and Dohme's biosimilar version of Lantus.
Two other biosimilar versions of Eli Lilly's osteoporosis drug Forteo (teriparatide) were also recommended for approval Friday.
Not all companies got good news from the EMA, however. Ultragenyx, a rare-disease drugmaker based in California, revealed it had withdrawn its marketing application for aceneuramic acid prolonged release after the EMA said Phase 2 data offered insufficient evidence to support an approval. Aceneuramic acid is one of Ultragenyx's three Phase 3 programs.