Attention Biotech Investors: Mark Your Calendar For September PDUFA Dates

August was a disappointing month as far as regulatory decisions are concerned. The FDA turned down several applications, citing different reasons.

Notable among the candidates that received the thumbs down were rheumatoid arthritis investigational therapy filgotinib sponsored by Gilead Sciences, Inc. GILD and BioMarin Pharmaceutical Inc.'s BMRN investigational gene therapy for hemophilia A.

That said, the month saw approvals of eight new molecular entities, taking the total for the year to 38.

Here are the key PDUFA dates scheduled for September.

Bristol-Myers Squibb Awaits Nod For Blood Cancer Maintenance Therapy

• Company: Bristol-Myers Squibb Co's BMY
• Type of Application: NDA
• Candidate: CC-486
• Indication: Acute myeloid leukemia
• Date: Sept. 3

CC-486 is an investigational oral hypomethylating agent, for the maintenance treatment of adult patients with acute myeloid leukemia, who achieved complete remission or complete remission with incomplete blood count recovery.

The FDA announced the acceptance of NDA for priority review May 1.

Can Mallinckrodt's Kidney Failure Drug Sneak Through FDA Hurdle After a Close Adcom Verdict?

• Company: Mallinckrodt PLC MNK
• Type of Application: NDA
• Candidate: terlipressin
• Indication: hepatorenal syndrome type 1, or HRS-1
• Date: Sept. 12

Terlipressin, which has been approved in countries outside of the U.S. and Canada, is a potent vasopressin analogue selective for V1 receptors. It's currently being investigated in the U.S. and Canada for HRS-1, an acute and life-threatening syndrome involving acute kidney failure in people with cirrhosis.

FDA's Cardiovascular and Renal Drugs Advisory Committee, which met July 15 to discuss the NDA, voted to recommend approval by a narrow margin, with eight voting for and seven against.

Eton Eyes Regulatory Nod For Partnered Eye Inflammation Drug

• Company: Bausch Health Companies Inc BHC and Eton Pharmaceuticals Inc ETON
• Type of Application: NDA
• Candidate: EM-100
• Indication: allergic conjunctivitis.
• Date: Sept. 15

EM-100 is an OTC formulation of ketotifen preservative-free ophthalmic solution that is being evaluated for allergic conjunctivitis. In February 2019, Eton out-licensed U.S. commercial rights for the ophthalmic solution to Bausch.

Following a regulatory filing by Bausch last year, the application was rejected by the FDA in July 2019. Bausch filed an amendment to the application in December. The FDA accepted the amendment and assigned a PDUFA date of Aug. 10.

Citing a proprietary name change submission, the FDA extended EM-100's goal date to Sept. 15.

Related Link: 2 Moderna Analysts On Chances Of COVID-19 Vaccine Approval, Upcoming Catalysts

GlaxoSmithKline Seeks Label Expansion For Asthma Drug

• Company: GlaxoSmithKline plc GSK
• Type of Application: sBLA
• Candidate: mepolizumab
• Indication: hypereosinophilic Syndrome
• Date: Sept. 26 (estimated)

Mepolizumab, which is sold under the trade name Nucala, was first approved in 2015 as a maintenance treatment of asthma in patients age 12 years and older. It's a humanized interleukin-5 antagonist monoclonal antibody produced by recombinant DNA technology in Chinese hamster ovary cells. Nucala reduces severe asthma attacks by reducing the levels of blood eosinophils- a type of white blood cell that contributes to the development of asthma.

GlaxoSmithKline is currently seeking label expansion for the biological treatment to include a third indication, namely hypereosinophilic syndrome.

The company announced May 27 granting of priority review for the application. Assuming filing was done 60 days prior to the acceptance date and giving a six-month review period, the goal date is likely to be Sept. 26.

Will Aquestive's Seizure Drug Clear The Regulatory Hurdle?

• Company: Aquestive Therapeutics Inc AQST
• Type of Application: NDA
• Candidate: libervant (AQST-203)
• Indication: seizure clusters
• Date: Sept. 27

Aquestive announced FDA acceptance of Libervant buccal film NDA filing Feb. 10.

"If approved by the FDA, Libervant will be the first oral diazepam-based therapy approved for management of seizure clusters in the population of 1.2 million refractory epilepsy patients. Libervant was designated by the FDA as an orphan drug in November 2016," the company said in the release announcing the acceptance of the regulatory application.

Eton's In-licensed Pediatric Adrenal Insufficiency Drug Awaits FDA Clearance

• Company: Eton Pharma
• Type of Application: NDA
• Candidate: Alkindi Sprinkle
• Indication: pediatric adrenal insufficiency
• Date: Sept. 29

Eton acquired U.S. marketing rights to Alkindi Sprinkle from AIM-listed Diurnal Group plc in late March. Alkindi Sprinkle, a taste neutral sprinkle or granule formulation of hydrocortisone, is being evaluated as a replacement therapy for pediatric adrenal insufficiency, including congenital adrenal hyperplasia in patients from birth to less than 17 years of age.

The product, which is approved in Europe, is to be available strengths 0.5mg, 1mg, 2mg, and 5mg to provide pediatric atients with optimal precision and flexibility.

Eton estimates the market opportunity of Alkindi Sprinkle to be $100 million annually.

Month-End Goal Date For Mesoblast's Stem Cell Therapy

• Company: Mesoblast limited MESO
• Type of Application: BLA
• Candidate: remestemcel-L (MSC-100-IV)
• Indication: steroid-refractory acute graft versus host disease, or SR-aGVHD, in children.
• Date: Sept. 30

Remestemcel-L, which goes by the trade name Ryoncil, is Mesoblast's lead product candidate. It's an investigational therapy comprising culture-expanded mesenchymal cells, derived from the bone marrow of an unrelated donor.

There are currently no FDA-approved treatments in the U.S. for children under 12 with SR-aGVHD, a potentially life-threatening complication of an allogeneic bone marrow transplant for blood cancer, Mesoblast said in a release.

FDA's Oncologic Drugs Advisory Committee, which met in mid-August, voted nine to one in favor that the available data support the efficacy of Ryoncil in pediatric patients with SR-aGVHD.

Adcom Meetings

FDA's Pulmonary-Allergy Drugs Advisory Committee is scheduled to discuss GlaxoSmithKline plc's GSK sNDA for Trelegy Ellipta, a fixed-dose combination of fluticasone furoate, umeclidinium, and vilanterol inhalation powder oral inhalation, for use in patients with chronic obstructive pulmonary disease.