Skip to main content


United States Food and Drug Administration (FDA) has actively given the green light to a number of efforts over the past few days to promote therapies for rare or serious diseases that have largely unmet medical needs. Here’s a look at today’s approvals.

SAGE Therapeutics received an FDA Fast Track designation for its potential Huntington’s disease (HD) drug. SAGE-178 is in development as a treatment for HD after studies have shown it to help improve cognitive performance, especially in terms of executive functioning.

“We believe that improving cognitive function is one of the main avenues for maintaining quality of life in HD and remains an area of ​​significant unmet medical need. The FDA’s Fast Track designation is an important step in the development of SAGE-718, as it provides opportunities to collaborate with the FDA to continue the clinical development and future regulatory review of SAGE-718 for the treatment of HD ” said Jim Doherty, Ph.D., director of research at Sage.

The pneumocide was also allowed a Fast Track designation for opelconazole, its drug candidate for invasive pulmonary aspergillosis. The latter also received the designations of an orphan drug and of production qualified as an infectious disease.

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, provides incentives for drug makers to find and create treatments for serious bacterial fungal and bacterial infections resistant to antibiotics.

“The FDA’s decision to grant the Opelconazole Orphan Drug, Fast Track, and QIDP designations recognizes the serious unmet medical need associated with API and is an important step in our efforts to develop a treatment for API. rare and debilitating, with less than 50% of patients meeting primary care standards well, ”commented Daniel Burgess, President and CEO of Pulmocide.

In addition to the two drugs mentioned, the FDA given a Fast Track designation for BridgeBio Pharma BBP-418, a treatment option under development for girdle muscular dystrophy type 2i (LGMD2i). The company is reportedly preparing to advance its Phase II clinical trial for BBP-418 in patients with genetically confirmed LGMD2i. If successful, BBP-418 could be the first approved treatment for the disease.

“We hope the designation will allow us to address this unmet medical need by allowing us to potentially deliver our drug to patients more quickly,” said Douglas Sproule, MD, M.Sc., Chief Medical Officer of ML Bio Solutions , the company BridgeBio which develops the drug.

Other notable approvals that the FDA gave in the week include fast-track approval for by BeiGene BRUKINSA (Zanubrutinib) for the treatment of relapsed or refractory marginal zone lymphoma in adult patients who have received at least one anti-CD20 regimen.

This too allowed Emergency use authorization for Cepheid Xpert Xpress CoV-2 / Flu / RSV plus, a rapid molecular diagnostic test to detect COVID-19, influenza A, influenza B, and respiratory syncytial virus (RSV.

Quanterix has also received emergency use authorization for its Simoa SARS-CoV-2 N protein antigen test to include nasal swab and saliva sample testing.

During this time, Therapeutic magenta was given Authorization of a new investigational drug (IND) for MGTA-117, which is considered a treatment for patients with relapsed or refractory acute myeloid leukemia (AML) and myelodysplastic syndromes (MDS).

Neurogene has also obtained 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 it green light for more than 100 requests for generic drugs with a competitive generic therapy designation to cover a wide range of products and therapeutic areas. The effort is aimed at making a previously tight-knit therapeutic world more open and accessible to all patients seeking highly effective and inexpensive alternatives to brand name drugs.

Leave a Reply