The U.S. Food and Drug Administration (FDA) has recently issued key approvals to several drugs that can treat rare or serious diseases, according to BioSpace.
Here are some key drug approvals the FDA has issued recently:
SAGE-178
SAGE-178, an experimental drug developed by Sage Therapeutics, has received a Fast Track designation from the FDA for the treatment of Huntington’s disease (HD), a progressive brain disorder that causes uncontrolled movements, emotional problems, and loss of thinking ability (cognition).
Jim Doherty, Chief Research Officer at Sage, said, “We believe that improving cognitive function is one of the core paths to maintaining the 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.”
Opelconazole
UK-based company Pulmocide also received a Fast Track designation for its experimental drug opelconazole for invasive pulmonary aspergillosis, a respiratory infection caused by a type of mold (fungus).
Pulmocide CEO Daniel Burges said, “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 the first-line standard of care.”
BBP-418
The FDA granted a Fast Track designation to BBP-418, an experimental treatment option for Limb-girdle Muscular Dystrophy Type 2i (LGMD2i). California-based BridgeBio Pharma is developing the drug to treat LGMD21, an autosomal recessive disorder.
Dr. Douglas Sproule of BridegBio said, “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.”
Others
Last week, the FDA granted accelerated approval for BeiGene’s Brukinsa (Zanubrutinib) for the treatment of relapsed or refractory marginal zone lymphoma in adult patients.
The agency also granted Emergency Use Authorization (EUA) for Xpress CoV-2/Flu/RSV plus, developed by Cepheid, which is a rapid molecular diagnostic test to detect COVID-19, Flu A, Flu B, and respiratory syncytial virus (RSV), according to BioSpace.
Massachusetts-based Quanterix also received EUA for its Simoa SARS-CoV-2 N protein antigen test that includes nasal swab and saliva sample testing.
Earlier this month, the FDA announced the approval of more than 100 generic drug applications with a Competitive Generic Therapy designation.
At the time, Dr. Sally Choe of the FDA, said, “This week, the FDA reached the milestone of approving more than 100 generic drug applications with a Competitive Generic Therapy (CGT) designation. This achievement highlights the success of the Competitive Generic Therapy program, which was designed to encourage the development and marketing of generic drugs for products with little to no competition.”
