Attention Biotech Investors: Mark Your Calendar For November PDUFA Dates

The loaded Prescription Drug User Fee Act (PDUFA) calendar for October produced mixed outcomes.

PDUFA dates are key binary events for biotech stocks that can move the needle in a big way. These are the dates by which the Food and Drug Administration is required to announce its decision regarding approvability/non-approvability of drugs.

ChemoCentryx, Inc.'s CCXI avacopan was the only new molecular entity approved during the month. NMEs are new drug products containing an active ingredient that is marketed for the first time in the U.S. Avacopan, which is to go by the brand name Tavneos, has been approved for treating small blood vessel damage.

Also approved in the month were an application seeking label expansion for Gilead Sciences, Inc.'s GILD CAR T-cell therapy Tecartus for use in advanced lymphoblastic leukemia and Oyster Point Pharma, Inc.'s OYST OC-01 (varenicline) nasal spray for treating dry eye disease.

Among the disappointments were Omeros Corporation's OMER narsoplimab as a treatment option for hematopoietic stem cell transplant-associated thrombotic microangiopathy, Eyenovia, Inc.'s EYEN MydCombi for use as a pupil dilation agent for eye examination and United Therapeutics Corporation's UTHR Tyvaso DPI (inhaled treprostinil) for pulmonary arterial hypertension.

Here are the key PDUFA catalysts for November.

Eton Hopes For Positive Verdict On Seizure Drug Following 3-Month Delay

• Company: Eton Pharmaceuticals, Inc. ETON
• Type of Application: new drug application
• Candidate: topiramate oral solution
• Indication: seizures
• Date: Nov. 6

Eton's topiramate oral solution is being evaluated as a treatment option for three indications – as a monotherapy for partial-onset or primary general tonic-clonic seizures in patients two years age and older, an adjunctive treatment of partial-onset seizures, including seizures associated with Lennox-Gastaut syndrome in patients two years of age and older, and as preventative treatment of migraine in patients 12 years of age and older.

The original PDUFA date of Aug. 6 was extended by three months to provide the regulator with additional time to review a amendment to the application by Azurity Pharma. Azurity had purchased topiramate, along with two other neurology candidates from Eton. Eton is eligible receive a $5 million milestone payment, contingent on approval of the candidate, and also royalties on sales. The new PDUFA date is Nov. 6.

Can Second Time Be Charm For Liquidia

• Company: Liquidia Corporation LQDA
• Type of Application: NDA
• Candidate: LIQ861
• Indication: pulmonary arterial hypertension
• Date: Nov. 7

LIQ861 is an investigational, inhaled dry powder formulation of treprostinil designed using Liquidia's novel print technology and engineered with the goal of enhancing deep-lung delivery of treprostinil in PAH patients by means of a convenient, palm-sized dry powder inhaler.

The original NDA was rejected with a complete response letter in late November, with the FDA seeking additional information and clarification on chemistry, manufacturing and controls data regarding the drug product and device compatibility.

Following a resubmission of the application in May, the FDA accepted the application, deeming it as a complete class 2 response.

Will BioMarin Successfully Take Its Short-limbed Dwarfism Drug Past The Finish Line?

• Company: BioMarin Pharmaceutical Inc. BMRN
• Type of Application: NDA
• Candidate: Vosoritide
• Indication: achondroplasia
• Date: Nov. 20

The regulatory application for vosoritide was accepted for review in November 2020 with a PDUFA date of Aug. 20. Subsequently, in April, the company said the FDA considered the submission of two-year results from the Phase 3 extension study as a major amendment to the application, and extended the PDUFA date to Nov. 20.

Vosoritide is an investigational, once daily injection for treating children with achondroplasia, the most common form of disproportionate short stature in humans.

Related Link: The Week Ahead In Biotech: Earnings Pick Up Pace, Eton Awaits Seizure Drug Approval, Kidney Conference, More IPOs In The Pipeline

Will FDA Tow In Line With Adcom Verdict On Takeda's CMV Antiviral Drug

• Company: Takeda Pharmaceutical Company Limited TAK
• Type of Application: NDA
• Candidate: maribavir (TAK-620)
• Indication: cytomegalovirus infection in transplant recipients
• Date: Nov. 23

Maribavir is being evaluated for the treatment of refractory cytomegalovirus infection and disease with genotypic resistance to ganciclovir, valganciclovir, foscarnet or cidofovir in transplant recipients. FDA's Antimicrobial Drugs Advisory Committee, which met in early October, voted unanimously, recommending approval of the treatment candidate.

Aadi Seeks Approval For Drug to Treat Rare Tumors

• Company: Aadi Bioscience, Inc. AADI
• Type of Application: NDA
• Candidate: Fyarro
• Indication: advanced malignant PEComa
• Date: Nov. 26

Fyarro is Aadi's lead product that is being evaluated for PEComa, which are rare tumors that form in the soft tissues of the stomach, intestines, lungs, female reproductive organs, and genitourinary organs. The FDA accepted the NDA for priority review on July 26.

Can Fennec's Pedmark Cross The FDA Hurdle In its Second Try?

• Company: Fennec Pharmaceuticals Inc. FENC
• Type of Application: NDA
• Candidate: Pedmark (sodium thiosulfate)
• Indication: chemotherapy-induced hearing loss in Children
• Date: Nov. 27

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. Following the receipt of a complete response letter in August 2020, Fennec resubmitted the application, which was accepted by the FDA in late June 2021.

'Go' or ‘No-Go' For VBI's Hepatitis B Virus Shot

• Company: VBI Vaccines Inc. VBIV
• Type of Application: BLA
• Candidate: Sci-B-Vac
• Indication: hepatitis B virus
• Date: Nov. 30

Sci-B-Vac is a 3-antigen prophylactic hepatitis B vaccine candidate for the prevention of infection caused by all known subtypes of the hepatitis B virus in adults. It is the only 3-antigen hepatitis B vaccine, comprising S, pre-S1, and pre-S2 surface antigens of the hepatitis B virus, and is approved for use and commercially available in Israel.

VBI's BLA was accepted for review on Feb. 2.

Hepatitis B is one of the world's most significant infectious disease threats with more than 290 million people infected globally. An estimated 780,000 people die each year from complications of chronic HBV such as liver decompensation and hepatocellular carcinoma.

Amryt Has Tryst With FDA For Rare Skin Disorder Drug

• Company: Amryt Pharma plc AMYT
• Type of Application: NDA
• Candidate: Filsuvez (Oleogel-S10)
• Indication: epidermolysis bullosa
• Date: Nov. 30

Filsuvez is a potential treatment for the cutaneous manifestations of junctional and dystrophic epidermolysis bullosa, a rare genetic skin disorder affecting young children and adults for which there is currently no approved treatment. Filsuvez has been selected as the brand name for Oleogel-S10.

Related Link: Moderna To Supply Additional COVID-19 Vaccine Doses To COVAX Alliance

BeyondSpring Seeks Nod For Combo Treatment In Chemotherapy-Associated Toxicity

• Company: BeyondSpring Inc. BYSI
• Type of Application: chemotherapy-induced neutropenia
• Candidate: plinabulin + G-CSF Combination
• Indication: NDA
• Date: Nov. 30

Plinabulin. in combination with granulocyte colony-stimulating factor, is being studied for the prevention of chemotherapy-induced neutropenia
Each year in the U.S., 110,000 patients receiving chemotherapy are hospitalized after developing CIN, a severe side effect that increases the risk of infection with fever.

CTI BioPharma Awaits Word On Rare Blood Cancer Drug

• Company: CTI BioPharma Corp. CTIC
• Type of Application: NDA
• Candidate: pacritinib
• Indication: myelofibrosis patients with severe thrombocytopenia
• Date: Nov. 30

The FDA accepted the regulatory application for priority review on June 1. Pacrotinib, if approved, is expected to addresses the unmet medical need of myelofibrosis patients with severe thrombocytopenia.