EMA gives thumbs up to key drugs from Novartis, Alexion, Vertex and more
→ The EMA’s Committee for Medicinal Products for Human Use has adopted positive opinions for a slate of drugs, issuing recommendation approvals that the EU will very likely follow. They include:
- Enerzair (QVM149), one of the key drugs that Novartis CEO Vas Narasimhan has tapped as a potential blockbuster. The asthma treatment comprises a long-acting beta-agonist, or LABA, called indacaterol acetate, a long-acting muscarinic antagonist in glycopyrronium bromide, and the corticosteroid mometasone furoate. The product would be administered once-daily through an inhaler.
- Alexion’s Ultomiris for atypical hemolytic uremic syndrome (aHUS) — for both complement inhibitor treatment-naïve patients and anyone who’s been responding to Soliris. If approved, it would be the first and only long-acting C5 inhibitor for the ultra-rare but serious indication in Europe, R&D chief John Orloff noted. In two ongoing single-arm, open-label studies, Ultomiris helped adult patients and children normalize certain blood parameters and improve kidney function at an interim analysis.
- A label expansion for Vertex’s CF therapy Kalydeco, which has previously been the center of heated pricing debates with payers. An OK here would cover children and adolescents between 6 months and 18 years old who have the R117H mutation on the CFTR gene. “Today’s announcement is important for young people with CF, as early intervention and treatment of this devastating and progressive disease is key to keeping patients healthier longer,” Carmen Bozic, who’s been promoted to the chief medical officer role as Reshma Kewalramani takes the helm, in a statement.
- CHMP has recommended for approval a subcutaneous form of J&J’s multiple myeloma drug Darzalex. The formulation uses Halozyme’s technology to deliver the drug in five minutes as opposed to over several hours by IV infusion, the biotech said.
→ Achieve Life Sciences, a Canadian biotech developing a drug for nicotine cessation, has executed a $1.9 million private placement for its stock, at $0.33 per share. The company has had mixed success in late-stage clinical trials, but concerns about patent ability of its product — which has long been available in parts of Europe — and coming generics for Chantix have killed its stock price.