Most FDA decisions scheduled for September yielded positive results, but there were stray disappointments as well. Seven new molecular entities, or NMEs, were approved during the month, taking the total NME approvals for the year to 24. This is far less than the 40 approvals received by the same time last year.
NMEs are considered a measure of innovation in drug research, as they contain active moieties or ingredients that haven’t been previously approved by the Food and Drug Administration.
Among the positive verdicts, bluebird bio, Inc.’s BLUE $3 million gene therapy Skysona was approved for treating a rare neurodegenerative disorder in boys and Amylyx Pharmaceuticals, Inc. AMLX scored a win for its amyotrophic sclerosis treatment Revance Therapeutics, Inc.’s RVNC Botox rival Daxxify was approved for treating frown lines.
Spectrum Pharmaceuticals, Inc. SPPI had a mixed month, as its Rolvedon was approved for treating chemotherapy-induced neutropenia, but its lung cancer drug did not get the backing of an FDA panel.
Here are the key PDUFA dates scheduled for October:
Will Theratech Score A Win For A More Convenient Formulation Of Its HIV Drug?
Company: Theratechnologies, Inc. THTX
Type of Application: supplemental biologic license application
Date: Oct. 3
In the fourth quarter of 2021, the company filed the sBLA for intravenous push mode of administration of its Trogarzo for treating human immunodeficiency virus type 1, or HIV-1. Trogarzo was first approved by the FDA in March 2018 to treat adult patients with multidrug-resistant HIV. In the quarter that ended on May 31, the drug generated sales of $7.852 million for Theratech.
Alnylam On Course For Snagging Label Expansion For Oxlumo?
Company: Alnylam, Inc. ALNY
Type of Application: supplemental new drug application
Indication: hyperoxaluria Type 1
Date: Oct. 6
Oxlumo, which goes by the generic name lumasiran, an RNAi therapeutic, is being evaluated for the reduction of plasma oxalate in patients with advanced hyperoxaluria type 1 – a rare disorder that mainly affects the kidneys due to the accumulation of a substance called oxalate, which is normally filtered through the kidneys and excreted in the urine.
This therapy was first approved in 2020 for lowering urinary oxalate labels in pediatric and adult patients. In the second quarter, it fetched the company $15 million in sales, down only 2% sequentially.
Can Third Time Be Charm For scPharma
Company: scPharmaceuticals, Inc. SCPH
Type of Application: NDA
Indication: worsening heart failure due to congestion
Date: Oct. 8
Furoscix is a proprietary, subcutaneously-delivered furosemide solution, that is being evaluated as an outpatient alternative for the treatment of worsening congestive heart failure. This is scPharma's third try for Furoscix following a complete response letter in 2018 and 2020.
’Go’ or ‘no-Go’ for Amicus’s Pome Disease Treatment?
Company: Amicus Therapeutics, Inc. FOLD
Type of Application: BLA
Candidate: cipaglucosidase alfa
Indication: Pompe disease
Date: Oct. 29
AT-GAA is an investigational 2-component therapy that consists of cipaglucosidase alfa, administered in conjunction with miglustat, which is yet to be approved by the FDA. The 2-component therapy is being evaluated for an inherited lysosomal disorder called Pome disease.
The disease can be debilitating and is characterized by severe muscle weakness that worsens over time. It ranges from a rapidly fatal infantile form with significant impacts on heart function to a more slowly progressive, late-onset form primarily affecting skeletal muscle. It is estimated that Pompe disease affects approximately 5,000 to 10,000 people worldwide.
Supernus Hopes For No Hiccups In Parkinson Disease Drug Approval
Company: Supernus Pharmaceuticals, Inc. SUPN
Type of Application: NDA
Candidate: apomorphine infusion device (SPN-830)
Indication: OFF episodes in Parkinson’s disease
Date: Early October
Supernus’s initial application seeking approval for SPN-830 was shot down with a refuse-to-file letter in November 2020, citing an insufficiently completed application to allow a substantiative review. Following a resubmission, the FDA accepted the application and assigned a PDUFA date sometime in early October.
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AstraZeneca Knocks At FDA Altar For Liver Cancer Combo Treatment
Company: AstraZeneca, plc. AZN
Type of Application: BLA/sBLA
Candidate: Tremelimumab + Imfinzi
Indication: Unresectable hepatocellular carcinoma
The FDA accepted AstraZeneca’s BLA for tremelimumab for priority review, supporting the indication of a single priming dose of the anti-CTLA4 antibody added to Imfinzi, for treating patients with unresectable hepatocellular carcinoma. A sBLA has also been submitted for Imfinzi for the indication.
This novel dose and schedule of the combination is called the STRIDE regimen - Single Tremelimumab Regular Interval Durvalumab. HCC, according to the company, is the most common type of liver cancer, and about 26,000 people in the U.S. are afflicted by advanced, unresectable HCC each year.
Merck-AstraZeneca Await Approval For Prostate Cancer Como Therapy
Company: Merck & Co., Inc. MRK & AstraZeneca
Type of Application: sNDA
Candidate: Lynparza in combination with Abiraterone and Prednisone or Prednisolone
Indication: prostate cancer
The application was accepted and granted priority review by the FDA for the treatment of adult patients with metastatic castration-resistant prostate cancer.
FDA’s Cardiovascular and Renal Drugs Advisory Committee will meet on Oct. 26 to discuss GlaxoSmithKline plc’s GSK NDA for hypoxia-inducible factor prolyl hydroxylase inhibitor daprodustat tablets as a treatment option for anemia resulting from chronic kidney disease.
The Oncologic Drugs Advisory Committee is scheduled to meet on Oct. 28 to discuss the BLA submitted by Y-mAbs Therapeutics, Inc. YMAB for its 1311-omburtamab solution, injectable formulation, for treating neuroblastoma with central nervous system/leptomeningeal metastases.
On Oct. 31, the Endocrinologic and Metabolic Drugs Advisory Committee will deliberate on Ispen S.A.’sIPSEY palovarotene capsules for the proposed indication of heterotopic ossification in adults and children. It is a condition in which muscle tissue and connective tissue such as tendons and ligaments are gradually replaced by bones.
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