The FDA is wrapping up business before the end of the year and making a flurry of approvals. The agency OK'd three new drugs Friday, with all of the green-lighted therapies sanctioned for combating relatively rare ailments and securing orphan drug status.
In terms of industry impact, the biggest of the three approvals went to Ariad Pharmaceuticals ($ARIA) for its new leukemia drug ponatinib, which could become a blockbuster product. (Read FierceBiotech's article from Friday for more details.)
The drug giants Novartis ($NVS) and GlaxoSmithKline ($GSK) were the two other recipients of FDA stamps on new drugs Dec. 14. The U.S. regulator approved GSK's raxibacumab, a monoclonal antibody from Human Genome Sciences, for treating patients poisoned with anthrax. And Novartis secured the FDA's authorization to market a therapy for the rare condition known as Cushing's disease.
Glaxo's approval stands out because the FDA for the first time blessed a monoclonal antibody for the market under a review pathway that allows for approval based on efficacy studies solely in non-human animals. Obviously, anthrax poisoning is a rare and deadly occurrence, and it'd be unethical to expose patients to anthrax to study the therapy. Its efficacy was proven in studies involving monkeys and rabbits, with its safety tested in 326 healthy humans.
Rockville, MD-based Human Genome Sciences--which GSK acquired in July for $3.6 billion--discovered raxibacumab and developed the therapy with support from the U.S. Biomedical Advanced Research and Development Authority (BARDA) and the FDA. The government has funded research of new treatments against anthrax in response to the threat of attacks with the lethal substance.
With Signifor, Novartis says the company has the first approved therapy in the U.S. for the underlying mechanism of Cushing's disease, which involves an overproduction of the hormone cortisol and can lead to complications such as obesity, diabetes and increased risk of infection.
The FDA has prided itself on being the first to approve the majority of new therapies, but the European Commission beat the agency on the approval of Signifor and authorized use the drug for Cushing's in April.
- here's a release on the Signifor approval
- see a release on raxibacumab
- check out Bloomberg's article on Novartis' win