FDA Grants Key Approvals to Potential Rare and Serious Disease Treatments


The U.S. Food and Drug Administration (FDA) has been actively greenlighting a number of efforts over the last few days to push therapies for rare or serious diseases that have largely unmet medical needs. Here's a look at today's approvals. 

SAGE Therapeutics received a Fast Track designation from the FDA for its potential Huntington's Disease (HD) drug. SAGE-718 is being developed as a treatment for HD after studies showed it helped improve cognitive performance, especially in terms of executive functioning.

"We believe that improving cognitive function is one of the core paths to maintaining quality of life in HD and remains an area of significant unmet medical need. The FDA Fast Track Designation is an important milestone in the development of SAGE-718, as it provides opportunities to engage collaboratively with the FDA to further clinical development and future regulatory review of SAGE-718 for the treatment of HD," said Jim Doherty, Ph.D., chief research officer at Sage. 

 Pulmocide was also granted a Fast Track designation for opelconazole, its drug candidate for invasive pulmonary aspergillosis. The latter also received Orphan Drug and Qualified Infectious Disease Production designations. 

Invasive pulmonary aspergillosis is a rare and debilitating disease, with less than 50 percent of patients responding well to first-line interventions. A QIDP designation, in particular, offers incentives to drug makers to find and create treatments for serious fungal and antibiotic-resistant bacterial infections. 

“The FDA’s decision to grant opelconazole Orphan Drug, Fast Track and QIDP designations acknowledges the serious unmet medical need associated with IPA and is a significant milestone in our effort to develop a treatment for IPA, a rare and debilitating disease, with less than 50% of patients responding well to first line standard of care,” commented Daniel Burgess, the chairman and chief executive officer of Pulmocide. 

In addition to the two drugs mentioned, the FDA gave a Fast Track designation to BridgeBio Pharma's BBP-418, a treatment option being developed for Limb-girdle Muscular Dystrophy Type 2i (LGMD2i). The company is reportedly preparing to advanced its Phase II clinical trial for BBP-418 in patients with genetically confirmed LGMD2i. If this succeeds, BBP-418 could be the first approved treatment for the disease. 

"We are hopeful the designation will allow us to address this unmet medical need by allowing us to potentially deliver our medicine to patients more quickly," said Douglas Sproule, M.D., M.Sc., the chief medical officer of ML Bio Solutions, the BridgeBio company that is developing the drug.

Other notable approvals that the FDA has given in the week include Accelerated Approval for BeiGene's BRUKINSA (Zanubrutinib) for the treatment of relapsed or refractory marginal zone lymphoma in adult patients who have received at least one anti-CD20-based regimen.

It also granted Emergency Use Authorization for Cepheid's Xpert Xpress CoV-2/Flu/RSV plus, a rapid molecular diagnostic test to detect COVID-19, Flu A, Flu B, and respiratory syncytial virus (RSV.

Quanterix also received Emergency Use Authorization for its Simoa SARS-CoV-2 N protein antigen test to include nasal swab and saliva sample testing. 

Meanwhile, Magenta Therapeutics was given Investigational New Drug (IND) clearance for MGTA-117, which is being considered as a treatment for patients with relapsed or refractory acute myeloid leukemia (AML) and myelodysplastic syndromes (MDS).

Neurogene was also granted IND clearance for its NGN-101 for the potential treatment of Batten Disease.

The week's approvals, among several others, come just two weeks after the FDA announced that it greenlit over 100 generic drug applications with a Competitive Generic Therapy designation to cover a wide range of products and therapeutic areas. The endeavor is meant to make a previously tight-knit therapy world more open and accessible to all patients looking for highly effective and low-cost alternatives to branded drugs. 

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