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FDA grants orphan drug status to Allogene’s BCMA-targeted treatment for multiple myeloma

The decision follows the RMAT designation granted by the FDA in April

Allogene HQ

The US Food and Drug Administration (FDA) has granted orphan-drug designation (ODD) to Allogene Therapeutics’ ALLO-715.

ALLO-715, an AlloCAR T therapy targeting B-cell maturation antigen (BCMA), is a potential novel treatment for multiple myeloma and other BCMA-positive malignancies.

Phase 1 data from the ALLO-715 UNIVERSAL trial demonstrated for the first time that an allogeneic CAR T therapy directed at BCMA can achieve clinical responses while eliminating the need for bridging therapy or delays in treatment associated with manufacturing.

Initial results were presented in December 2020 at an oral session of the American Society of Hematology annual meeting. In April 2021, ALLO-715 was granted Regenerative Medicine Advanced Therapy (RMAT) designation by the FDA.

“We are pleased to have received ODD for ALLO-715 just months after the FDA granted RMAT designation. These designations from the FDA underscore the importance of bringing this important therapeutic option to patients with multiple myeloma,” said Rafael Amado, executive vice president of research & development and chief medical officer of Allogene. “We look forward to presenting the next update from our UNIVERSAL trial by the end of 2021 and providing additional insight into the potential of our allogeneic cell therapy platform.”

The FDA grants ODD to drugs or biologics intended to treat a rare disease or condition, affecting fewer than 200,000 individuals in the US. ODD designation allows pharma companies to utilise development incentives, such as tax credits for clinical testing, prescription drug user fee exemptions and seven-year marketing exclusivity in the event of regulatory approval.

The UNIVERSAL trial will continue to evaluate ALLO-715, including in combination with nirogacestat, a gamma secretase inhibitor from SpringWorks Therapeutics and in consolidation therapy.

The next clinical update is expected in late 2021.

Article by
Bryony Andrews

13th August 2021

From: Regulatory



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