Attention Biotech Investors: Mark Your Calendar For October PDUFA Dates

Following a mixed month for regulatory approvals, biotech investors now turn toward a month of plenty as far as Food and Drug Administration decisions are concerned.

Four new molecular entities, or NMEs, were approved in September. Seagen Inc. SGEN and Genmab's GMAB antibody-drug conjugate Tivdak for the treatment of metastatic cervical cancer and Takeda Pharmaceutical's TAK Exkivity for the treatment of EGFR exon 20 insertion–positive non–small cell lung cancer were among the NME approvals for the month.

Incyte Corporation INCY received twin regulatory nods this month.

On the other hand, Calliditas Therapeutics AB CALT was in for a disappointment as the drug regulator announced a three-month extension in the review period for its Nefecon as a treatment option for primary IgA Nephropathy.

Verrica Pharmaceuticals Inc.'s VRCA viral skin infection treatment candidate was rejected by the FDA.

Here are the key PDUFA catalysts for the unfolding month.

Click here to access Benzinga's FDA Calendar

Gilead Eyes Additional Approval For Tecartus In Severe Leukemia Patients

Company: Gilead Sciences, Inc. GILD
Type of Application: supplemental biologic license application
Candidate: Tecartus
Indication: blood cancer
Date: Oct. 1

Tecartus (brexucabtagene autoleucel) is a CAR T-cell therapy that's being evaluated in adult patients with relapsed or refractory B-cell precursor acute lymphoblastic leukemia. Gilead acquired the asset through its Kite acquisition. It's currently indicated for the treatment of adult patients with relapsed or refractory mantle cell lymphoma.

Can Chemocentryx's Avacopan Find a Smooth Sailing Following An Extended Review?

Company: ChemoCentryx, Inc. CCXI
Type of Application: new drug application
Candidate: avacopan
Indication: ANCA-Associated Vasculitis
Date: Oct. 7

Avacopan is an orally-administered small molecule that employs a targeted mode of action in the treatment of ANCA-associated vasculitis and other complement-driven autoimmune and inflammatory diseases.

ANCA-associated vasculitis is a systemic disease in which over-activation of the complement pathway further activates neutrophils, leading to inflammation and destruction of small blood vessels. This leads to organ damage and failure, mainly the kidney, and is fatal if not treated.

The FDA accepted the avacopan NDA for review on Sept. 17, 2020, and set an original PDUFA goal date of July 7. An FDA panel that reviewed the NDA in early May issued a split verdict. The regulator finally extended the review period by three months.

Avadel Hopes To Take Its Sleep Disorder Drug Past The Finish Line

Company: Avadel Pharmaceuticals plc AVDL
Type of Application: NDA
Candidate: FT218
Indication: sleep disorder
Date: Oct. 15

FT218, an investigational, once-nightly formulation of sodium oxybate for the treatment of excessive daytime sleepiness and cataplexy in adults with narcolepsy.

If approved, FT218 will be the first and only once-nightly oxybate medication, a significant advancement to the twice-nightly regimen that has been required for nearly 20 years, the company had said in a statement.

FT218 will be approved by the PDUFA date, and its sales can grow to over $300 million in 2023, and generate sales in the $400 million+ range in the outer years on a non-risk-adjusted basis given its more convenient once-nightly dosing vs other sodium oxybates, Needham analyst Ami Fadia said in a recent note.

‘No' Or ‘No-go' For United Therapeutics' Dry Powder Formulation Of Pulmonary Arterial Hypertension Drug?

Company: United Therapeutics Corporation UTHR
Type of Application: NDA
Candidate: Tyvaso DPI (inhaled treprostinil)
Indication: Pulmonary arterial hypertension
Date: Oct. 15

United Therapeutics is developing Tyvaso DPI under a collaboration and license agreement with MannKind Corporation MNKD.

Tyvaso DPI incorporates the dry powder formulation technology and Dreamboat inhalation device technology used in MannKind's Afrezza inhalation powder product, which was approved by the FDA in 2014. If approved, Tyvaso DPI is expected to provide a more convenient method of administration as compared with traditional nebulized Tyvaso therapy.

Oyster Point Pharma Awaits Nod For Dry Eye Disease Drug

Company: Oyster Point Pharma, Inc. OYST
Type of Application: NDA
Candidate: OC-01 (varenicline) nasal spray
Indication: dry eye disease
Date: Oct. 17

OC-01 nasal spray is a highly selective cholinergic agonist being developed as a multi-dose preservative-free nasal spray to treat the signs and symptoms of dry eye disease. Dry eye disease, a chronic, progressive condition that impacts more than 30 million people in the U.S., is a multifactorial condition of the ocular surface characterized by disruption of the tear film.

Dry eye disease affects quality of life, as it can cause persistent stinging, scratching, burning sensations, sensitivity to light, blurred vision and eye fatigue.

The NDA was accepted for review on March 2, with a decision-by date of Oct. 17. The company is planning a U.S. launch in the fourth quarter, contingent on FDA approval.

Omeros Hopes For No Further Hiccups In Narsoplimab Approval

Company: Omeros Corporation OMER
Type of Application: BLA
Candidate: narsoplimab
Indication: hematopoietic stem cell transplant-associated thrombotic microangiopathy
Date: Oct. 17

Omeros' narsoplimab is its lead MASP-2 inhibitor that targets the lectin pathway of complement. It's also in multiple late-stage clinical development programs focused on other complement-mediated disorders, including IgA nephropathy, atypical hemolytic uremic syndrome and COVID-19.

Transplant-associated thrombotic microangiopathy is an endothelial damage syndrome that is increasingly identified as a complication of both autologous and allogeneic hematopoietic cell transplantation in children.

The original PDUFA date of July 17 was extended by three months due to the additional time sought by the FDA to review information provided by the company in response to a request by the agency.

Ocular Seeks Label Expansion For Dextenza

Company: Ocular Therapeutix, Inc. OCUL
Type of Application: sNDA
Candidate: Dextenza
Indication: allergic conjunctivitis
Date: Oct. 18

Dextenza is being evaluated for the treatment of ocular itching associated with allergic conjunctivitis. It was previously approved for the treatment of ocular inflammation and pain following ophthalmic surgery.

It's a corticosteroid intracanalicular insert placed in the punctum, a natural opening in the inner portion of the lower eyelid, and into the canaliculus and is designed to deliver dexamethasone to the ocular surface for up to 30 days without preservatives.

Will Sanofi-Regeneron's Dupixent Get Another Label Expansion?

Company: Sanofi SNY & Regeneron Pharmaceuticals, Inc. REGN
Type of Application: sBLA
Candidate: Dupixent
Indication: asthma in children
Date: Oct. 21

Dupixent is being evaluated as an add-on treatment for children aged 6 to 11 years with uncontrolled moderate-to-severe asthma. Dupixent is currently approved as an add-on treatment for patients with uncontrolled moderate-to-severe asthma aged 12 and older with elevated eosinophils or oral corticosteroid dependent asthma.

Roche Has a Date With FDA For Its Novel Therapeutic For Retinal Disorder

Company: Roche Holding AG RHHBY
Type of Application: BLA
Candidate: Port Delivery System with ranibizumab, or PDS
Indication: neovascular or wet age-related macular degeneration
Date: Oct. 23

PDS is a permanent refillable eye implant designed to continuously deliver a customized formulation of ranibizumab over a period of months, potentially reducing the treatment burden associated with frequent eye injections.

If approved, PDS, according to the company, would be a first-of-its-kind therapeutic approach, offering people living with nAMD an alternative to frequent eye injections of anti-vascular endothelial growth factor, the current standard of care.

Eyenovia's Pupil Dilation Agent Up Before FDA Altar

Company: Eyenovia, Inc. EYEN
Type of Application: NDA
Candidate: MydCombi
Indication: Pupil dilation agent for eye examination
Date: Oct. 28

MydCombi is a fixed combination pupil dilation agent for potential use in eye exams. Eyenovia noted that if approved, MydCombi would be the first microdosed ocular therapeutic applied with a high precision smart delivery system, the Optejet.

Can Second Time Be Charm For ANI Pharma?

Company: ANI Pharmaceuticals, Inc. ANIP
Type of Application: sNDA
Candidate: purified Cortrophin gel
Indication: multiple indications
Date: Oct. 29

Purified Cortrophin gel is a purified adrenocorticotropic hormone previously approved in the U.S. for multiple indications including multiple sclerosis, rheumatoid arthritis, and nephrotic syndrome.

The FDA originally approved Cortrophin Gel in 1954 for the treatment of several autoimmune conditions. The usage of the therapy was discontinued in the 1980s. ANI Pharma acquired it from Merck & Co. Inc. MRK in early 2016 and worked on validating the therapy and modernizing its manufacturing and control processes to align with current FDA standards.

The original regulatory application submitted by ANI Pharma was rejected by the FDA in April 2020 due to concerns with the quality of information provided about the therapy's chemistry, manufacturing, and control processes. Following the company's resubmission, the FDA accepted it for review in late August.

Can Clearside Biomedical & Bausch Succeed In Second Try For Macular Edema Drug?

Company: Clearside Biomedical, Inc. (LSD & Bausch Health Companies Inc. BHC
Type of Application: NDA
Candidate: Xipere (triamcinolone acetonide)
Indication: macular edema associated with uveitis.
Date: Oct. 30

Xipere is a proprietary suspension of the corticosteroid triamcinolone acetonide formulated for administration to the suprachoroidal space that is being investigated for the treatment of macular edema associated with uveitis.

The initial NDA submitted by the duo was issued a complete response letter in Oct. 2019, and a resubmission of the application was done in early May. The FDA accepted the resubmitted application in early June and deemed it as a Class 2 resubmission.

Adcom Calendar

FDA's Antimicrobial Drugs Advisory Committee will discuss Takeda's NDA for maribavir oral tablets for the treatment of adults with post-transplant cytomegalovirus infection and/or disease, including infections resistant. The meeting is scheduled to be held Oct. 7, between 9 a.m. and 5 p.m.

The Oncologic Drugs Advisory Committee is scheduled to deliberate on Oncopeptides AB ONPPF's NDA for Pepaxto (melphalan flufenamide) for injection, in combination with dexamethasone, for the treatment of adult patients with relapsed or refractory multiple myeloma who have received at least four prior lines of therapy and whose disease is refractory to at least one proteasome inhibitor, one immunomodulatory agent, and one CD38-directed monoclonal antibody. The meeting will be held Oct. 28, between 10:30 a.m. and 3 pm.

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