Bluebird bio Seeks Back-to-Back Nods, 'Go' or 'No-Go' For Revance's Botox Rival, Amylyx' Twin Hurdles And More: September's Key PDUFA Catalysts Biotech Investors Must Know

After August’s mixed tally for Food and Drug Administration approvals, investors turn to a new month, which has a fairly loaded calendar.

New molecular entity approvals drew a blank in August. NMEs are those active moieties that have not been previously approved by the FDA. This should be a cause of concern, as NME approvals are considered a measure of innovation in drug research.

The fortunate ones that could take their drugs past the finish line included bluebird bio, Inc. BLUE, Johnson & Johnson JNJ, and Axsome Therapeutics, Inc. AXSM.

ACADIA Pharmaceuticals, Inc. ACAD faced rejection for expanding the label of its Nuplazid to be able to treat Alzheimer’s disease psychosis. CorMedix, Inc.’s CRMD defencath application was shot down by the FDA for a second time. The review period for Amicus Therapeutics, Inc.’s FOLD Pompe disease treatment was extended.

The Prescription Drug User Fee Act, or PDUFA, date is the period by which the FDA is required to rule on an application submitted by a sponsor company.

Here are the key PDUFA catalysts for the upcoming month:

Can Vertex’s Cystic Fibrosis Drug Snag a Label Expansion

Company: Vertex Pharmaceuticals Incorporated VRTX
Type of Application: supplemental new drug application
Candidate: Orkambi
Indication: cystic fibrosis in children.
Date: Sept. 4

Orkambi, a combination of ivacaftor and lumacaftor, was first approved for cystic fibrosis in July 2015. The company is now seeking to expand the label of the drug to include children 12 months to less than 24 months old.

Cystic fibrosis is a rare genetic disease that leads to a build-up of thick mucus in the lungs and other organs. It is caused by a defect in or absence of the protein – “cystic fibrosis transmembrane conductance regulatory protein,” due to mutations in the CFTR gene.

Orkambi fetched Vertex sales of $772 million in 2021, down from $908 million a year ago. A potential label expansion could help reinvigorate sales.

Revance Frown Lines Treatment Awaits FDA Clearance

Company: Revance Therapeutics, Inc. RVNC
Type of Application: biologic license application
Candidate: daxibotulinumtoxinA
Indication: glabellar lines
Date: Sept. 8

DaxibotulinumtoxinA, or Daxi, is a next-gen neuromodulator product that is being evaluated to treat moderate to severe glabellar lines, aka frown lines, and is touted as a potential Botox rival. The original PDUFA date of Nov. 25, 2020, was not met due to COVID delays in inspecting manufacturing sites.

The FDA issued a complete response letter – a communication from the FDA regarding non-approvability of the treatment, in October 2021, citing issues with manufacturing sites. The company’s resubmitted BLA was accepted for review by the FDA in late April, with the new PDUFA date.

Can Second Time Be Charm For Spectrum Pharma

Company: Spectrum Pharmaceuticals, Inc.’s SPPI
Type of Application: BLA
Candidate: eflapegrastim
Indication: chemotherapy-induced neutropenia
Date: Sept. 9

Eflapegrastim, which is to be marketed under the trade name Rolontis, is a long-acting granulocyte-colony stimulating factor. It's a novel molecule for the treatment of neutropenia, with the potential to compete in a worldwide market that exceeds $6 billion, Spectrum said in an investor presentation.

Neutropenia is a condition where the neutrophil count is very low in the blood and it can be caused due to diseases that damage the bone marrow by certain medications, including chemotherapy.

Like Revance, the FDA’s decision on Spectrum Pharma’s Rolontis application was delayed from the original PDUFA date of September 2020. A year later, the drug regulator outrightly rejected the application, citing issues with the manufacturing sites in South Korea. The FDA accepted the resubmitted application and communicated the new PDUFA date.

Bristol-Myers Squibb Looks to Add To Its New Drug Launch Tally For 2022

Company: Bristol-Myers Squibb Company BMY
Type of Application: NDA
Candidate: deucravacitinib
Indication: plaque psoriasis
Date: Sept. 10

Bristol-Myers Squibb's deucravacitinib, if approved, would be the first tyrosine kinase 2 inhibitor approved for the treatment of any disease. Phase 3 data supporting the NDA submission demonstrated the drug’s superior efficacy relative to Amgen, Inc.’s AMGN Otezla.

Otezla, which has been approved for multiple indications, generated sales of $2.3 billion for Amgen in 2021.

Bristol-Myers Squibb has been brisk with its first-in-class drug approvals this year, and if deucravacitinib is approved by the deadline, it would mark the third such drug approval of the year for the company, Fierce Biotech reported.

ObsEva Hopes For Uterine Fibroid Drug Nod In US After EU Approval

Company: ObsEva SA OBSV
Type of Application: NDA
Candidate: linzagolix
Indication: heavy menstrual bleeding
Date: Sept. 13

Linzagolix provides flexible dosing options for the management of heavy menstrual bleeding associated with uterine fibroids in premenopausal women.

“A dosing option without hormonal ABT would be welcomed by the significant number of women who either have contraindications to or a personal preference to avoid the use of estrogen-based therapies, while also providing a dosing option for women in whom hormonal ABT is indicated,” the company’s chief medical officer Elizabeth Garner said in a statement.

The European Commission granted marketing authorization for linzagolix in June, and in the region it would be sold under the brand name Yselty,

bluebird Braces For Double Delight

Company: bluebird bio
Type of Application: BLA
Candidate: eli-cel
Indication: cerebral adrenoleukodystrophy
Date: Sept. 16

Bluebird bio is coming off the successful clearance for Zynteglo, its cell-based gene therapy, for the treatment of beta-thalassemia in adult and pediatric patients requiring red blood cell transfusions. The approval secured in mid-August kickstarted a nice upward momentum in the stock.

If approved, eli-cel will be the first and only gene therapy for the treatment of early active CALD, a rare neurodegenerative disease that primarily affects young children and leads to irreversible loss of neurologic function and death.

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Will It Be Smooth Sailing For Heron’s Nausea Drug?

Company: Heron Therapeutics, Inc. HRTX
Type of Application: NDA
Candidate: HTX-019
Indication: post-operative nausea and vomiting
Date: Sept. 17

Heron sees a significant market opportunity for HTX-019, going by about 39 million surgical procedures done annually, where people are at risk for PONV. The regulatory application submitted in Nov. 2021 was accepted for review with a PDUFA goal date of Sept. 17.

Regeneron Seeks Another Label Expansion For Cancer Drug

Company: Regeneron Pharmaceuticals, Inc. REGN
Type of Application: Libtayo in combination with chemotherapy
Candidate: sBLA
Indication: Lung cancer
Date: Sept. 19

Libtayo, a PD-1 inhibitor jointly developed by Regeneron and Sanofi SNY, has already been approved for three indications – two types of skin cancer and as a monotherapy option for lung cancer.

The companies are now attempting to get a fourth approval for the treatment, this time in combination with chemotherapy as a first-line treatment for advanced non-small cell lung cancer patients.

Will FDA Hear Out Fennec’s Argument For Its Hearing Loss Drug?

Company: Fennec Pharmaceuticals Inc. FENC
Type of Application: NDA
Candidate: Pedmark
Indication: ototoxicity
Date: Sept. 23

Pedmark is a unique formulation of sodium thiosulfate, and is being evaluated for the prevention of ototoxicity induced by cisplatin chemotherapy in patients one month to less than 18 years of age with localized, non-metastatic, solid tumors.

Fennec’s original application was handed with a CRL in November 2021 on the grounds of manufacturing issues. A resubmission was made and the FDA accepted it for review in April.

Will Amylyx’ Wait For Maiden FDA Nod End?

Company: Amylyx Pharmaceuticals, Inc.’s AMLX
Type of Application: NDA
Candidate: sodium phenylbutyrate/taurursodiol
Indication: amyotrophic lateral sclerosis
Date: Sept. 29

An FDA panel, which met in March, voted 6-4 that the single Phase 2 CENTAUR trial and the open-label extension study did not conclude that the investigational asset is effective in treating ALS, and the drug regulator later announced an extension to the review period. This pushed the PDUFA date from June 29 to Sept. 29. A new Adcom meeting was convened subsequently.

Given the drug is the only pipeline compound in Amylyx’ portfolio, it becomes all the more important for the company to push it past the finish line. On a positive note, the company snagged its first-ever nod in June, when Health Canada approved the combo treatment for ALS.

Sanofi-Regeneron On Cusp Of Getting Another Approval For Blockbuster Drug

Company: Regeneron/Sanofi
Type of Application: sBLA
Candidate: Dupixent
Indication: prurigo nodularis
Date: Sept. 30

Dupixent is Sanofi-Regeneron combo’s blockbuster anti-inflammatory drug, which has been approved for multiple indications. The two companies are now knocking on the FDA’s door for getting approval for Dupixent in prurigo nodularis, a chronic inflammatory skin disease that causes extreme itch and skin lesions.

If greenlighted, Dupixent would be the first and only medicine approved specifically to treat prurigo nodularis in the U.S.

Adcom Calendar:

The Peripheral and Central Nervous System Drugs Advisory Committee has convened a meeting on Sept. 7, between 12 p.m. and 6:30 p.m., to discuss Amylyx’ NDA for sodium phenylbutyrate/taurursodiol powder for oral suspension, for the treatment of ALS.

The FDA’s Oncologic Drugs Advisory Committee Meeting will occur on Sept. 22-23, both days between 9 a.m. and 1:15 p.m., to discuss Spectrum Pharma’s NDA for poziotinib tablets for the treatment of patients with previously treated, locally advanced or metastatic non-small cell lung cancer harboring HER2 exon 20 insertion mutations.