Today, a brief rundown of news from the Food and Drug Administration and Insmed, as well as updates from Sanofi, Oryon Cell Therapies, Sarepta Therapeutics and GSK that you may have missed.
The Food and Drug Administration is seeking industry feedback on the "national priority" voucher program it's been using to accelerate drug reviews. The agency is accepting public comments ahead of a June 12 meeting, during which it will look for opinions on the selection process, eligibility criteria, review procedures and other aspects of the program. Since launching the initiative last year, the FDA has awarded 18 vouchers, rejected a drug from one recipient and approved another four. Two of those clearances were issued within two months of a submission. The program has drawn criticism from some lawmakers as well as, reportedly, agency staff claiming it's become a tool for the White House to influence drug approvals.
Insmed's drug Arikayce met its main objective in a late-stage study involving people with a lung disease triggered by a kind of chronic infection. According to Insmed, Arikayce meaningfully reduced respiratory symptoms compared to a placebo in patients who have this "MAC" lung illness but haven't yet received antibiotics. The roughly 3.1-point difference between the two groups on symptom scores, as measured by a patient-reported questionnaire, were "somewhat smaller than expected," wrote RBC Capital Markets analyst Leonid Timashev. Still, the RBC team thinks "the drug has done enough" to support an expanded approval and the outcome should remove an "overhang" that has seen Insmed shares regain some of the value they've lost this year. The company's stock price climbed by double digits in early trading Monday.
Sanofi has licensed a three-pronged immune drug from biotechnology startup Kali Therapeutics. The French pharmaceutical company said Monday it will hand over $180 million in upfront and near-term payments, and potentially $1.05 billion in total for rights to the therapy, which is codenamed KT501 and has the potential to treat a "broad range" of B cell-mediated autoimmune conditions. KT501 is a trispecific antibody that targets the proteins CD3, CD19 and BCMA. It's currently being evaluated in a first-in-human study in people with rheumatoid arthritis.
Oryon Cell Therapies emerged from stealth Monday with former longtime Acorda Therapeutics CEO Ron Cohen as its top executive. The startup is developing personalized cell therapies designed to replace the neurons that degenerate in brain disorders like Parkinson’s disease. Its lead program is currently in human testing in Parkinson’s, and early data were presented at a medical meeting over the weekend. Oryon says it’s raised $42 million A so far, including most recently a $21 million Series A tranche that saw participation from Neuro.VC and Byers Capital.
Sarepta Therapeutics will submit requests to the FDA by the end of April to convert the “accelerated” approvals covering two of its Duchenne muscular dystrophy drugs into traditional clearances. Sarepta said last year that the “totality of the evidence” supported standard approvals for the medicines, Vyondys 53 and Amondys 45, even though they both failed their main objectives in a confirmatory study. U.S. regulators have now agreed that the company can use real-world evidence as well as data from that trial in its submission. Still, the FDA’s acceptance doesn’t “materially change” the risk of a rejection, given the negative findings reported last year and the fact that the current FDA hasn’t been “very amenable to regulatory flexibility,” wrote Leerink Partners analyst Joseph Schwartz.
The FDA on Thursday approved Lynavoy, a drug GSK developed for a kind of severe itching associated with the liver disease primary biliary cholangitis. The clearance makes Lynavoy the first medicine available in the U.S. for the condition, which is believed to affect some 89% of people with PBC, according to GSK. Even so, the drug won’t be launched by GSK. The British pharmaceutical company agreed last month to license Lynavoy’s full rights to Italian pharmaceutical company Alfasigma in a deal potentially worth up to nearly $700 million. That pact was signed to “sharpen GSK’s focus” on other liver disease therapies, top scientist Tony Wood said at the time. It included a $100 million payout for GSK upon U.S. approval.
