The FDA is heading into fall with a few Prescription Drug User Fee Act (PDUFA) dates. Here’s a look at this week’s upcoming action.
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The U.S. Food and Drug Administration is heading into fall with a few new Prescription Drug User Fee Act (PDUFA) dates. Here’s a look at this week’s upcoming action.
Obseva’s Linzagolix for Uterine Fibroids
Obseva, based in Geneva, Switzerland, has a target action date of Sept. 13 for a New Drug Application (NDA) for linzagolix to treat uterine fibroids.
The drug is the first and only GnRH receptor antagonist that provides flexible dosing options, including a low dose for women who can’t or do not want to take hormones.
The NDA was built on data from two Phase III PRIMROSE trials (1 and 2). The studies evaluated two dosing regimens, 100mg once daily and 200mg once daily, alone or in combination with hormonal add-back therapy (ABT). This is for the treatment of heavy menstrual bleeding linked to uterine fibroids. The NDA was made up of positive 24-week treatment data from both trials and supportive data from Week 52 and 76-week post-treatment follow-up.
Bluebird bio’s Eli-Cel for Cerebral Adrenoleukodystrophy
Bluebird bio has a target action date of Sept. 16 for its gene therapy, elivaldogene auto excel (eli-cel), for cerebral adrenoleukodystrophy (CALD).
Eli-cel has tended to be linked with bluebird’s other gene therapy, Zynteglo (betibeglgene autotemcel) for beta-thalassemia, which the FDA approved on Aug. 18. Both had PDUFA goal dates three months earlier, which were pushed back when the agency requested additional material. They both use a Lenti-D lentiviral vector.
“We all share a responsibility to be diligent for patients as we progress this novel field,” said bluebird CEO Andrew Obenshain in a Jan. 18 statement.
CALD is a progressive, irreversible and eventually fatal disease that primarily affects young children. It is a neurologic disorder that causes rapid loss of neurological function after the initial onset of the disease. Almost half of the patients who aren’t treated die within five years of the start of symptoms.
The accumulation of very long-chain fatty acids (VLCFA) in the adrenal cortex and the brain and spinal cord white matter destroys myelin, the protective sheath of the brain nerve cells responsible for thinking and muscle control.
Eli-cel utilizes ex vivo transduction with the Lenti-D lentiviral vector (LVV) to insert functional copies of the ABCD1 gene into a patient’s own hematopoietic stem cells (HSCs). This allows patients to manufacture the ALD protein, which is believed to help break down very long-chain fatty acids.
Heron’s HTX-019 for Prevention of Postoperative Nausea and Vomiting
Heron Therapeutics has a target action date of Sept. 17 for its NDA for HTX-019 for the prevention of postoperative nausea and vomiting (PONV).
The drug is an IV injectable emulsion formulation designed to deliver aprepitant, the active ingredient in EMEND capsules, directly. This is the only Neurokinin 1 receptor antagonist (NK1 RA) approved in the U.S.
EMEND was approved by the FDA for oral dosing within three hours before induction of anesthesia for surgery. In a Phase I trial, HTX-019 32mg as a 30-second IV injection was bioequivalent to oral aprepitant 40mg.
“In a recent Cochrane Meta-Analysis, aprepitant was found to be the most effective agent for PONV prevention with activity similar to two-drug combinations,” said Barry Quart, Pharm.D., chairman and CEO of Heron, in a November 2021 statement. “In fact, the use of oral aprepitant has grown by almost 80% in the past three years without any promotional efforts.”
