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.

Novocure GmbH secured U.S. FDA approval for Optune Lua, a wearable device that delivers alternating electric fields or tumor treating fields that kill cancer cells. Indicated for use with PD-1/PD-L1 inhibitors or docetaxel in the second or subsequent line for the treatment of metastatic non-small-cell lung cancer following the failure of platinum-based regimens, the approval was driven by the significant improvement in overall survival rates seen in the LUNAR pivotal study for Optune Lua.

The U.S. FDA granted Momentis Surgical Ltd. 510(k) clearance for its Anovo robotic surgical platform for use in single site, abdominal access ventral hernia repairs. The U.S. regulatory agency also gave the greenlight to CMR Surgical Ltd.’s Versius system for use in cholecystectomy, in a sign of the growing market for smaller, more portable robotic devices to assist in minimally invasive procedures.

Seraxis Inc. has obtained IND clearance from the FDA for a phase I/II study of its novel islet replacement therapy SR-02 for type 1 diabetes. SR-02 is comprised of allogeneic pancreatic endocrine cell clusters that, when implanted to the omentum, form a functional endocrine pancreas outside of the native pancreas.

Borvo Medical Inc., a neurointerventional med-tech startup, reported U.S. FDA 510(k) clearance of its Endoport Vacuum Assisted Collection (EVAC) system, an alternative to traditional subdural hematoma (SDH) drainage methods. The EVAC system is expected to be available in early 2025 the company said.

The U.S. FDA’s device center disclosed its guidance ambitions for this new fiscal year – a list that includes the usual A and B lists for draft and final guidances. However, the agency now has an “under construction” list of guidance ambitions, the status of which is entirely reliant on the agency’s resources.

Genentech Inc. didn’t need to wait until Thanksgiving for the U.S. FDA to make up its mind. More than a month ahead of its PDUFA date, the agency approved the firm’s first-line breast cancer treatment, Itovebi (inavolisib), providing the oral therapy a place with other niched therapies from Astrazeneca plc and Novartis AG. Itovebi is to be combined with Pfizer Inc.’s palbociclib (Ibrance) and Faslodex (fulvestrant, Astrazeneca) for adults with endocrine-resistant, PIK3CA-mutated, hormone receptor (HR)-positive, HER2-negative, locally advanced or metastatic breast cancer.

“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. Peterson, a vice provost, senior associate dean and professor at the University of Texas Southwestern Medical Center, was one of 10 members of the U.S. FDA’s Cardiovascular and Renal Drugs Advisory Committee (CRDAC) who came to that conclusion. Six others had a different opinion. Regardless of which way they voted, the panelists attested to how difficult the decision was.