Mari Serebrov

Depending on how long it lasts, the dockworkers strike that hit all 14 ports on the U.S. East Coast and along the Gulf Oct. 1 could literally unload a world of hurt to patients and the biopharma/med-tech industries, as it will impact both U.S. imports and exports of life-saving drugs and technologies.

It’s well past time for the U.S. FDA to end its silence on what device patents can be listed in the Orange Book as part of a drug-device combination product, Sen. Bill Cassidy (R-La.) said in an Oct. 1 letter that took FDA Commissioner Robert Califf to task for letting the FTC do the FDA’s job.

Round 2 of the U.S. Medicare price negotiations officially began late Oct. 2 with CMS’ release of its final playbook and timeline, which build on lessons learned from the first round and comments on a draft negotiations guidance that was published in May. In response to the feedback CMS received, the second round will allow for more patient-focused sessions and offer increased engagement with the makers of the drugs selected for negotiations.

A growing foray of pharmacy benefit managers’ (PBMs) private labels into the U.S. biosimilar space is intensifying concerns about the antitrust aspects of PBMs’ vertical integration that has them serving as price negotiator, formulary setter, payer, group purchasing organization, pharmacy, provider and now drug "manufacturer."

Stealth Biotherapeutics Inc. had hoped the U.S. FDA would have approved its lead candidate, elamipretide, as the first treatment for Barth syndrome by now. Instead, it’s headed to a meet-up with the agency’s Cardiovascular and Renal Drugs Advisory Committee (CRDAC) Oct. 10. The discussion and vote at that meeting could be make-or-break for patients with the ultra-rare debilitating mitochondrial disease that has no approved therapies. “Barring support from CRDAC, the future of elamipretide for Barth syndrome in the U.S. is tenuous,"Stealth CEO Reenie McCarthy told BioWorld.

With confidence dropping in doing business with China-affiliated life sciences companies due to the Biosecure Act, one of the companies explicitly named in the U.S. legislation is fighting back. Raising the possibility of a constitutional challenge to the bill, Rade Drmanac, co-founder and chief scientific officer of Complete Genomics Inc., wrote to congressional leaders to urge them to remove the California-based subsidiary of MGI from the bill.

“This was worse than our national election,” Eric Peterson said as he explained his vote Oct. 10 concluding that Stealth Biotherapeutics Inc.’s elamipretide is effective in treating Barth syndrome, an ultra-rare mitochondrial disease that currently affects 129 males in the U.S.

With confidence dropping in doing business with China-affiliated life sciences companies due to the Biosecure Act, one of the companies explicitly named in the U.S. legislation is fighting back.

Coming on the heels of an advisory committee in which the U.S. FDA and its independent advisers grappled with trying to fit an ultra-rare disease development program into the confines of the agency’s “significant evidence” requirements, an Oct. 16 public meeting on a Rare Disease Innovation Hub the agency is setting up seemed like a welcome step in the right direction for rare disease patients, their caregivers and companies working in the space.

Insider trading isn’t always about profits. Sometimes it’s avoiding losses. That’s the basis of the U.S. SEC’s complaint against Matthew Groom, an information technology consultant to Spero Therapeutics Inc. Groom agreed Sept. 15 to a $28,000 settlement to resolve the complaint stemming from a trade of Spero shares that enabled him to avoid $13,000 in losses when news of the company’s downsizing and issues with its lead product became public two years ago.