FDA, BrainStorm confer regarding investigational ALS treatment

The FDA has conferred with BrainStorm Cell Therapeutics Inc. regarding NurOwn, an investigational technology platform developed to treat amyotrophic lateral sclerosis.

In a release, BrainStorm, which specializes in the development of adult stem cell therapies, said the primary objective of the meeting was to discuss key considerations for a Special Protocol Assessment (SPA) affecting a planned phase 3b registrational trial for NurOwn.

For an SPA, drug manufacturers may ask to meet with the FDA to reach agreement on the design and size of certain clinical trials to determine if they address scientific and regulatory requirements for a study that could support marketing approval.

The agreement indicates concurrence by FDA with adequacy and acceptability of specific critical elements of overall design for a study but does not indicate concurrence on every detail.

BrainStorm is expected to submit relevant documentation as outlined by the FDA to support the SPA, with the goal of securing the FDA’s agreement that critical elements of the overall study protocol design (eg, entry criteria, endpoints, planned analyses) will be sufficient for a study intended to support a future marketing application.

“Our meeting with the FDA provided us with valuable input on the phase 3b trial design, outlining clear steps forward,"” BrainStorm President and CEO Chaim Lebovits said in the release. “We are grateful for the FDA’s continued support and guidance and look forward to providing further updates once we have more clarity on an SPA.”