October proved to be a mixed month for the Food and Drug Administration’s regulatory decisions. Two new molecular entities, or NMEs, were approved during the month, bringing the tally for the year-to-date period to 28.
Among the NME approvals were Johnson & Johnson (NYSE: JNJ (NYSE:JNJ)) unit Janssen’s Tecvayli, a BCMA-targeting bispecific, for treating multiple myeloma in patients who have tried at least four prior lines of therapy. AstraZeneca (NASDAQ:AZN) plc’s (NASDAQ: AZN) tremelimumab-durvalumab combo received the nod for treating adult patients with inoperable hepatocellular carcinoma.
On the other hand, Amicus Therapeutics, Inc.'s (NASDAQ: FOLD) two-component drug for treating Pompe disease, characterized by severe muscle weakness, was rejected by the FDA.
Adcom decisions were mostly negative, with a panel postponing the meeting scheduled for discussing Ispen S.A.’s (OTC: IPSEY) palovarotene capsules to treat a condition in which muscle and connective tissue such as tendons and ligaments will be gradually replaced by bones. Shares of Y-mAbs Therapeutics, Inc. (NASDAQ: YMAB) plunged about 60% on Monday on a negative Adcom verdict.
A PDUFA, or Prescription Drug User Fee Act, date is the deadline fixed by the FDA to announce its verdict regarding the approvability or non-approvability of a drug. It is a binary catalyst that can trigger big stock swings.
Seagen Seeks Another Label Expansion For Its Blood Cancer Drug
- Company: Seagen, Inc. (NASDAQ: SGEN)
- Type of Application: supplemental biologic license application
- Candidate: Adcetris
- Indication: treatment-naïve Hodgkin’s lymphoma in adults
- Date: Nov. 16
Seagen is now seeking another expanded use for the drug. Adcetris is being evaluated in children and young adults with previously untreated, high-risk Hodgkin lymphoma.
In the recent quarter that ended in September, Adcetris fetched sales of $428 billion, up 17% year-over-year.
Can Travere Win FDA Nod For Rare Kidney Disorder Drug?
- Company: Travere Therapeutics, Inc. (NASDAQ: TVTX)/Ligand Pharmaceuticals, Inc. (NASDAQ: LGND)
- Type of Application: new drug application
- Candidate: sparsentan
- Indication: IgA nephropathy
- Date: Nov. 17
IgAN, aka Berger’s disease, is a rare kidney disorder characterized by the build-up of immunoglobin, which causes a breakdown of the normal filtering mechanisms, leading to blood and protein in the urine. It is the leading cause of primary glomerulonephritis, an end-stage kidney disease. More than 100,000 people in the U.S. are estimated to be suffering from the disorder.
Travere in-licensed the drug from Ligand, and an NDA submission by Travere will kick in a potential milestone payment of about $5.9 million for Ligand.
Provention Shrugs Off Hiccups And Looks To Take Its Diabetes Drug Past Finish Line:
- Company: Provention Bio, Inc. (NASDAQ: PRVB)/MacroGenics Inc. (NASDAQ: MGNX)
- Type of Application: BLA
- Candidate: teplizumab
- Indication: type 1 diabetes mellitus
- Date: Nov. 17
The company resubmitted the application in January 2022, and it was accepted for review in March with a new PDUFA goal date of Aug. 17. In late June, the regulator came back and said it is extending the review period by three months.
Will FDA Overlook Panel Verdict On Spectrum’s Lung Cancer Drug?
- Company: Spectrum Pharmaceuticals, Inc. (NASDAQ: SPPI)
- Type of Application: NDA
- Candidate: poziotinib
- Indication: lung cancer
- Date: Nov. 24
There is no FDA-approved therapy for this condition. The product has a Fast Track designation. An FDA panel, which reviewed the drug in late September, voted 9-4 that its benefits do not outweigh the risks.
‘Go’ or ‘no-Go’ For Apellis’ Eye Disorder Drug?
- Company: Apellis Pharmaceuticals, Inc. (NASDAQ: APLS)
- Type of Application: NDA
- Candidate: intravitreal pegcetacoplan
- Indication: geographic atrophy
- Date: Nov. 26
Apellis said, citing estimates, that GA affects more than five million people worldwide and is the leading cause of blindness. It also noted that there is no currently approved therapy for GA.
See also: During The Pandemic Tuberculosis Deaths Increased, Reversing Years Of Decline: WHO Report
ImmunoGen Knocks FDA Altar For Antibody-Drug Conjugate To Treat Ovarian Cancer
- Company: ImmunoGen, Inc. (NASDAQ: IMGN)
- Type of Application: BLA
- Candidate: mirvetuximab soravtansine monotherapy
- Indication: ovarian cancer
- Date: Nov. 28
The FDA accepted ImmunoGen’s BLA for review in late May.
SCYNEXIS Seeks To Expand The Use Of Vaginal Yeast Infection Drug:
- Company: SCYNEXIS, Inc. (NASDAQ: SCYX)
- Type of Application: sNDA
- Candidate: Brexafemme
- Indication: vulvovaginal candidiasis
- Date: Nov. 30
Can FDA Lift Y-mAbs Stock That Plunged On Adcom Verdict?
- Company: Y-mAbs Therapeutics Inc (NASDAQ: YMAB)
- Type of Application: BLA
- Candidate: omburtamab
- Indication: central nervous system/leptomeningeal metastasis from neuroblastoma.
- Date: Nov. 30
Nevertheless, the FDA will look into the application; therefore, the PDUFA action date of Nov. 30 stays intact. The regulator typically takes into account Adcom’s discussions while making its decision, but is not bound to go with it.
Adcom Calendar
- FDA's Pulmonary-Allergy Drugs Advisory Committee will discuss the NDA submitted by AstraZeneca and Bond Avillion 2 Development for a fixed dose combination of budesonide and albuterol sulfate metered dose inhaler. The proposed indication is as-needed treatment or prevention of bronchoconstriction and for the prevention of exacerbations in patients with asthma 4 years of age and older. The meeting is scheduled on Nov. 8, between 10 a.m. EDT and 4 p.m. EDT.
- The same committee will discuss Veru, Inc.’s (NASDAQ: VERU) request for emergency use authorization for sabizabulin oral capsule for treating SARS-CoV-2 infection in hospitalized patients with moderate to severe COVID-19 infection who are at high risk of acute respiratory distress syndrome. The meeting will be held on Nov. 9, between 10 a.m. ET and 4 p.m. ET.
- On Nov. 16, the Cardiovascular and Renal Drugs Advisory Committee will discuss the NDA for tenapanor hydrochloride tablets, submitted by Ardelyx, Inc. (NASDAQ: ARDX) for the control of serum phosphorus levels in adults with chronic kidney disease on dialysis. The Adcom will be held on Nov. 16, between 9:30 a.m. ET and 5 p.m. ET.
Read Next: Best Biotech Socks Right Now
© 2022 Benzinga.com. Benzinga does not provide investment advice. All rights reserved.