Cell Therapy News/Articles, page-16871

Some will argue its not strictly an allogeneic stem cell therapy. A rising tide floats all boats and the press is reporting as it is. Gamida recently dosed its first patient post FDA approval in April:

https://beststocks.com/gamida-cells-omisirge-receives-fda-approval-f/

"Gamida Cell Ltd. has made an exciting announcement on September 27, 2023, revealing that the very first patient has successfully undergone a groundbreaking stem cell transplant using Omisirge. This remarkable therapy is the only allogeneic stem cell treatment to have gained approval from the U.S. Food and Drug Administration (FDA) based on the results of a global randomized Phase 3 trial."

MSB is on its knees as a business and share, but the surrounding regulatory backdrop continues to improve.

(Don't feel too bad MSB'ers, Gamida's share price now almost as low as just prior to approval announcement - I used to own!!)

Meanwhile Brainstorm in ALS, got voted down - unsurprisingly:

https://medcitynews.com/2023/09/fda-advisory-committee-brainstorm-cell-therapeutics-als-stem-cell-therapy/

This at least put the FDA under the spot light again as it recently granted approval in two fairly unconvincingly trialed more conventional ALS treatments (sound familiar?). Arguably a bit of post-hoc data mining:

"But Brainstorm pointed to a post-hoc analysis of a subgroup"

Objectively, few gave Brainstorm and the thousands of patients petitioning the FDA much chance, and frankly MSB's case far more convincing and as we know efficacy not questioned by the FDA, according to Krause, but a few comments not irrelevant to MSB....:

"Caleb Alexander, professor of epidemiology and medicine at Johns Hopkins University’s Bloomberg School of Public Health, said the NurOwn data were hard to interpret and did not suggest efficacy, adding that Brainstorm’s application relies on a single study in which the results did not show efficacy. He acknowledged that there’s precedent for the FDA approving drugs based on a single pivotal study.

“Although there are some unfortunate examples, I think if you look at the majority of examples where FDA used a single trial, it’s been with very strong evidence to support the approvals,” Alexander said.

The FDA is not required to follow the votes of its advisory committees, but it does take committee discussions into account in its decisions. Peter Marks, director of the FDA’s Center for Biologics Evaluation and Research, concluded the meeting by acknowledging the patient testimony, adding that the agency will review the comments to the docket and the transcript of the advisory committee meeting.

“The FDA does hear the tremendous need here for effective therapies in this space, and that’s not lost on us,” Marks said."