Final ALS Drug Development Guidance Coming Soon

Regulatory NewsRegulatory News | 22 August 2019 |  By 

Acting US Food and Drug Administration (FDA) Commissioner Ned Sharpless on Thursday announced that the agency would finalize guidance on developing treatments for amyotrophic lateral sclerosis (ALS) by the end of September.

The 9-page draft guidance, first released in February 2018, incorporated parts of a draft guidance put together by the ALS Association, known for its viral "ice bucket challenge," which FDA said provided the agency "with scientific advice and insight into the disease that helped us advance our own draft guidance that provides our clear thinking on drug development in this area."

The ALS Association also held a workshop, attended by FDA officials, to discuss patient experience, benefits and risks, outcomes and study design.

“When patients are diagnosed with ALS, they are told they likely have two to five years to live. They will progressively lose all motor function and be completely dependent on caregivers for their survival. Clinical trial guidance, design, and execution must reflect this reality,” a workshop summary says.

The heightened anticipation around FDA’s release of final guidance on ALS drug development comes as the number of investigational products in trials is slowly growing. According to the nonprofit, “I Am ALS,” 11 clinical trials are currently enrolling ALS patients. The ALS Association also has a more extensive list of clinical trials. And although there are three approved treatments that may help patients with ALS, none can reverse, halt or prevent ALS.

According to BrainStorm, which is running one of the active trials, by the end of July, 170 patients were enrolled in its Phase 3 randomized, double blind, placebo-controlled, repeat-dose clinical trial for the experimental ALS treatment NurOwn. The company expects that by the second week of October, enrollment will be finalized with 200 patients. The company presented Phase 2 results at the Annual Meeting of the American Academy of Neurology in April 2018.

And if ALS patients cannot find an active trial to enroll in, they may be able to use FDA’s expanded access program or the Right-to-Try law.

Although for some experimental treatments, neither of those options are helpful. Startup Revalesio, for instance, is running early phase clinical trials with patients with ALS, but says its experimental drug “is not available for use under an expanded access program.”

Richard Garr, CEO of Beacon of Hope, a niche contract research organization dedicated to Right-to-Try treatment programs, previously told Focus that an ALS Right-to-Try program was expected to treat between 200 and 300 patients over the next 12 months.

Garr added via email on Thursday that the Right-to-Try programs are confidential but an ALS program is “up and running and have had very good response from the patient community. I can also tell you that I am ‘unveiling’ Beacon of Hope in mid-September at the Florida ALSA chapter’s Hope and Help Symposium, so all of the response so far, precedes the Company’s real outreach to the ALS community. Certainly I am confident that we will treat the 200 to 300 patients I mentioned in our last talk for the existing program, over a 12 month period.”

He also said he’s in “active discussions with other ALS treatment developers, but again, it is all confidential. But I can say publicly is that we are seeing great enthusiasm for exploring this path by companies, as well as on the patient side.”

ALS: Developing Drugs for Treatment

Editor's Note: Article updated to reflect that the ALS final guidance will be issued at the end of September, not August.


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