February PDUFA Catalysts Biotech Investors Must Know: Sanofi's Bleeding Disorder Drug, Regeneron's Twin Eylea Label Expansions, 3 Delayed Approvals And More

The year has started well for biopharma companies on the regulatory front, as most decisions handed out during January were positive. The Food and Drug Administration has already cleared four new molecular entities for the year. NME approvals assume importance as the metric is an indicator of innovation in drug research.

It was the case of a tale of two biotechs as far as Alzheimer’s drug research is concerned. Biogen, Inc. BIIB and partner Eisai Co. Ltd. ESALY received the nod for their second monoclonal antibody treatment. The drug, which is to go by the brand name Leqembi, received approval under the accelerated approval pathway.

On the other hand, the drug regulator denied accelerated approval for Eli Lilly & Co.’s LLY Alzheimer’s treatment candidate donanemab. Lilly now rests its hopes on data from a confirmatory trial due in the second quarter to file for full approval. Lilly had a consolation win with the approval of Jaypirca for treating a certain type of breast cancer.

Seagen, Inc. SGEN received the nod for expanding the label of its cancer drug Tukysa as a combo therapy to treat colorectal cancer and Merck & Co., Inc.’s MRK Keytruda succeeded in snagging another approval.

Now, here are the major PDUFA dates for the upcoming month:

GlaxoSmithKline Seeks Approval For Anemia Drug In Kidney Disease Patients Following Mixed Adcom Verdict

Company: GlaxoSmithKline plc GSK
Type of Application: new drug application, or NDA
Candidate: daprodustat
Indication: anemia of chronic kidney disease
Date: Feb. 1

The FDA accepted the application for review in April 2022 with a decision date of Feb. 1, 2023. Daprodustat is an oral hypoxia-inducible factor prolyl hydroxylase inhibitor for the potential treatment of anemia caused by chronic kidney disease. It is currently approved in Japan under the brand name Duvroq for patients with retinal anemia.

An FDA committee that discussed the drug ruled in favor of approving it to treat dialysis patients, but against its usage in non-dialysis patients.

Can Travere Convince FDA Of Kidney Inflammation Drug’s Liver Safety?

Company: Travere Therapeutics, Inc. TVTX
Type of Application: NDA
Candidate: sparsentan
Indication: IgA nephropathy
Date: Feb. 17

Travere faced a setback when the FDA extended the review period for the drug, pushing back the original PDUFA date of Nov. 17, 2023, by three months. The regulator asked the company to update its proposed Risk Evaluation Mitigation Strategy to include liver monitoring.

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. This causes blood and protein to pass through to 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 Pharmaceuticals, Inc. LGND.

Apellis Eyes Eye Disorder Drug Nod

Company: Apellis Pharmaceuticals, Inc. APLS
Type of Application: NDA
Candidate: intravitreal pegcetacoplan
Indication: geographic atrophy
Date: Feb. 26

Pegcetacoplan is a C3 therapy being evaluated for the treatment of GA secondary to age-related macular degeneration. Waltham, Massachusetts-based Apellis announced in July that the FDA accepted the pegcetacoplan application for review. In early November, the company said the decision date has been pushed back to give time for the FDA to review the two-year efficacy data from a pair of Phase 3 studies it was planning to submit.

GA, according to the company, affects more than five million people worldwide and is the leading cause of blindness. There is no currently approved therapy for the disorder.

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Can Cytokinetics’ Heart Failure Drug Succeed After Adcom Snub

Company: Cytokinetics, Inc. CYTK
Type of Application: NDA
Candidate: omecamtiv mecarbil tablets.
Indication: cardiovascular death and heart failure
Date: Feb. 28

Omecamtiv mecarbil is being evaluated for reducing the risk of cardiovascular death and heart failure events in patients with symptomatic chronic heart failure with reduced ejection fraction.

The FDA’s Cardiovascular and Renal Drugs Advisory Committee, which met in mid-December, voted 8 to 3 that the benefits of omecamtiv mecarbil do not outweigh its risks for the treatment of heart failure with reduced ejection fraction.

Cytokinetics has sold royalties on future worldwide sales of omecamtiv mecarbil to Royalty Pharma, Inc. RPRX.

Can Reata Put Setbacks Behind?

Company: Reata Pharmaceuticals, Inc. RETA
Type of Application: NDA
Candidate: omaveloxolone
Indication: Friedreich's ataxia
Date: Feb. 28

The FDA accepted the omaveloxolone application for priority review in May 2022 with a PDUFA date of Nov. 30, 2022. Following a mid-cycle communication meeting with the regulator, the company submitted additional data and analyses. These were considered major amendments and the FDA pushed back the PDUFA date to the current schedule.

Friedreich ataxia is a rare inherited disease that causes progressive damage to the nervous system and movement problems. There is currently no FDA-approved treatment for the disease.

Incidentally, Reata’s application seeking approval for bardoxolone, as a treatment option for chronic kidney disease caused by Alport’s Syndrome, was shot down by the FDA a year back.

Sanofi Seeks To Get Its Bleeding Disorder Biologics Past Finish Line

Company: Sanofi SNY
Type of Application: biologics license application
Candidate: efanesoctocog alfa
Indication: Hemophilia A
Date: Feb. 28

Sanofi and Sobi are collaborating on the development and commercialization of efanesoctocog alfa, which is being evaluated to treat hemophilia A, a rare genetic bleeding disorder. Hemophilia A occurs in about one in 5,000 male births annually, and more rarely in females.

Efanesoctocog alfa is a recombinant factor VIII therapy that is designed to extend protection from bleeds with once-weekly prophylactic dosing for people with hemophilia A.

Gilead’s Trodelvy Label Expansion

Company: Gilead Sciences, Inc. GILD
Type of Application: supplemental BLA
Candidate: Trodelvy
Indication: breast cancer
Date: February

Trodelvy is now being evaluated for treating a certain type of advanced breast cancer that cannot be surgically removed in patients who have received endocrine-based therapy and at least two additional systemic therapies.

Among others, Regeneron Pharmaceuticals, Inc. REGN has two PDUFA dates this month. The company is seeking label expansion for its Eylea in two indications. A decision on Eylea approval for retinopathy of prematurity in preterm infants is expected on Feb. 11. Also, the FDA will rule on a 16-week dosing regimen of Eylea in patients with diabetic retinopathy on Feb. 28.

Adcom Calendar

The FDA’s Oncologic Drug Advisory Committee is scheduled to meet on Feb. 9, between 11 a.m. and 5:30 p.m. EST, to discuss GlaxoSmithKline’s NDA for dostarlimab-gxly for injection as a monotherapy for patients with locally advanced, treatment-naïve mismatch repair deficiency/microsatellite instability-high rectal cancer.

The Nonprescription Drug Advisory Committee and the Anesthetic and Analgesic Drugs Products Advisory Committee would meet on Feb. 15, between 9 a.m. and 5:30 p.m. EST to discuss Emergent Biosolutions, Inc.’s EBS supplemental NDA for Narcan nasal spray for the nonprescription treatment of known or suspected opioid overdose.

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