Editor's note: This story has been corrected to reflect that the FDA deferred action on Amicus Therapeutics' Biologics License Application.
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 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 plc’s AZN tremelimumab-durvalumab combo received the nod for treating adult patients with inoperable hepatocellular carcinoma.
The FDA deferred action on Amicus Therapeutics, Inc.'s FOLD two-component drug for treating Pompe disease, characterized by severe muscle weakness, due to an inability to inspect a manufacturing site in China before the PDUFA goal date.
The site could not be inspected due to COVID-19 travel restrictions, according to a company press release.
“We are now one step away from the necessary approvals for AT-GAA in the U.S. We continue to believe this is a question of ‘when’ not ‘if’ AT-GAA will be approved and we will continue to work with great urgency to support the FDA’s completion of the final plant inspection necessary for approval so that this important new treatment option is made available for people living with Pompe disease in the United States,” Bradley Campbell, Amicus president and CEO, said in a statement.
Adcom decisions were mostly negative, with a panel postponing the meeting scheduled for discussing Ispen S.A.’s 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. 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. SGEN
- Type of Application: supplemental biologic license application
- Candidate: Adcetris
- Indication: treatment-naïve Hodgkin’s lymphoma in adults
- Date: Nov. 16
Adcetris was first approved by the FDA in 2011 for treating Hodgkin’s lymphoma and a rare lymphoma called systemic anaplastic large-cell lymphoma. It subsequently received five label expansions, with the last one secured in November 2018.
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. TVTX/Ligand Pharmaceuticals, Inc. LGND
- Type of Application: new drug application
- Candidate: sparsentan
- Indication: IgA nephropathy
- Date: Feb. 17, 2023
Travere announced the FDA’s acceptance of the regulatory application for priority review on May 16. The company said, if approved, sparsentan would be the first FDA-approved non-immunosuppressive treatment option for IgA nephropathy, or IgAN.
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. PRVB/MacroGenics Inc. MGNX
- Type of Application: BLA
- Candidate: teplizumab
- Indication: type 1 diabetes mellitus
- Date: Nov. 17
Provention Bio’s teplizumab was in-licensed from MacroGenics and it is being evaluated as an option for delaying Type 1 diabetes in at-risk individuals. In July 2021, the original application was handed down a complete response letter, which cited issues with the bridging study done comparing the drug and its predecessor drug from Eli Lilly & Co. LLY.
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. SPPI
- Type of Application: NDA
- Candidate: poziotinib
- Indication: lung cancer
- Date: Nov. 24
Poziotinib is a hydrochloride salt of poziotinib and is formulated as a tablet for oral administration. Spectrum’s NDA seeks approval of the drug for patients with previously-treated, locally-advanced or metastatic non-small cell lung cancer with HER2 exon 20 insertion mutations.
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. APLS
- Type of Application: NDA
- Candidate: intravitreal pegcetacoplan
- Indication: geographic atrophy
- Date: Nov. 26
Waltham, Massachusetts-based Apellis announced in July that the FDA accepted the application for review. Pegcetacoplan is a C3 therapy being evaluated for the treatment of GA secondary to age-related macular degeneration.
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.
ImmunoGen Knocks FDA Altar For Antibody-Drug Conjugate To Treat Ovarian Cancer
- Company: ImmunoGen, Inc. IMGN
- Type of Application: BLA
- Candidate: mirvetuximab soravtansine monotherapy
- Indication: ovarian cancer
- Date: Nov. 28
Mirvetuximab soravtansine is an investigational antibody drug conjugate that selectively kills cancer cells. It is being investigated as a monotherapy in patients with folate alpha-high platinum-resistant ovarian cancer who have been previously treated with one to three systemic treatments.
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. SCYX
- Type of Application: sNDA
- Candidate: Brexafemme
- Indication: vulvovaginal candidiasis
- Date: Nov. 30
Brexafemme is a novel oral antifungal approved for the treatment of vulvovaginal candidiasis, also known as vaginal yeast infection, in August. The company has now applied for approval of the same for an expanded indication. This time around, Brexafemme is being tried for the prevention of recurrent vulvovaginal candidiasis.
Can FDA Lift Y-mAbs Stock That Plunged On Adcom Verdict?
- Company: Y-mAbs Therapeutics Inc YMAB
- Type of Application: BLA
- Candidate: omburtamab
- Indication: central nervous system/leptomeningeal metastasis from neuroblastoma.
- Date: Nov. 30
FDA’s Oncologic Drugs Advisory Committee last week handed down a negative verdict on the application, which has dampened the odds of omburtamab scraping through. The committee voted 16 to 0 that the company had not provided sufficient evidence to conclude that omburtamab improves overall survival. The verdict sent traders scurrying away from the stock, resulting in the 60% plunge.
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.
- 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 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. 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.
The iShares Biotechnology ETF IBB was traded 0.94% higher on Tuesday to $129.78, according to Benzinga Pro data.
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Correction 11.2.2022: Travere Therapeutics’ assigned PDUFA date is Feb. 17, 2023, not Nov. 17 as originally reported.
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