Attention Biotech Investors: Mark Your Calendar For July PDUFA Dates

June was a month of plenty and also turned out to be a fruitful one from the perspective of Food and Drug Administration approvals. Regulatory reviews mostly produced positive verdicts, and rejections or no-decisions were few and far between.

New molecular entity approvals tapered off to merely two in June. SCYNEXIS, Inc.'s SCYX Brexafemme for treating vaginal yeast infection and Biogen Inc.'s BIIB Aduhelm for Alzheimer's were the two NMEs approved during the month.

NME approvals halfway through the year have exceeded the numbers for 2020 at the same time last year.

FDA's approval of Aduhelm stirred a controversy, given the poor data package that supported the approval, the broader label accorded to it and the higher pricing.

Among the not-so-fortunate candidates was Arimoclomol sponsored by Orphazyme A/S ORPH. This investigational drug to treat Niemann-Pick Disease Type C, a rare neurodegenerative disease, faced an outright rejection. The FDA did not communicate its decision on Takeda Pharmaceutical Company Limited's TAK TAK-721, which is being evaluated as a treatment option for eosinophilic esophagitis.

Here are the key PDUFA dates for July.

Provention Bio Waits For Sweet Tidings For Diabetes Drug After Mixed Regulatory Signals

Company: Provention Bio, Inc. PRVB
Type of Application: biologic license application
Candidate: teplizumab
Indication: type 1 diabetes mellitus
Date: July 2

Teplizumab is an investigational anti-CD3 monoclonal antibody being evaluated for the delay or prevention of clinical type 1 diabetes in at-risk individuals.

Provention announced FDA acceptance of the BLA for priority review in early January. In late April, the regulator warned of deficiencies in the regulatory application, deeming that the pharmacokinetic profiles of the company's candidate and Eli Lilly and Company's LLY original drug substance used in the bridging study are not comparable.

An Adcom panel that met on May 27 for reviewing the BLA voted in favor of approving the drug.

ChemoCentryx Up Against Odds With Its Drug to Treat Small Blood Vessel Damage

Company: ChemoCentryx, Inc. CCXI
Type of Application: new drug application
Candidate: avacopan
Indication: ANCA-Associated Vasculitis
Date: July 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 NDA for review on Sept. 17, 2020 and set a PDUFA goal date of July 7. An FDA panel that reviewed the NDA in early May issued a split verdict.

Raymond James analyst Steve Seedhouse said in a recent note he sees a 50/50 probability on avacopan approval. It's more likely a three-month extension is announced, he added.

Merck Aims to Be 'Better Late Than Never' In The Multi-strain Pneumococcal Vaccine Market

Company: Merck & Co., Inc. MRK
Type of Application: BLA
Candidate: V114
Indication: pneumococcal disease
Date: July 18

Merck's BLA for V114, an investigational 15-valent pneumococcal conjugate vaccine, for the prevention of invasive pneumococcal disease in adults 18 years of age and older, was accepted for priority review on Jan. 12.

Ligand Pharmaceuticals Incorporated LGND unit Pfenex had out-licensed its Pfenex Expression Technology Pseudomonas-based recombinant protein expression technology for the production of specific proteins to be used in the development of the vaccine candidate.

Rival Pfizer Inc. PFE recently received approval for Prevnar 20, a 20-valent Pneumococcal conjugate vaccine for the prevention of invasive disease and pneumonia in adults ages 18 years and older.

Will Albireo Get The Nod For Drug to Treat Itching In Inherited Liver Disease Patients?

Company: Albireo Pharma, Inc. ALBO
Type of Application: NDA
Candidate: odevixibat
Indication: pruritus
Date: July 20

The FDA accepted the odevixibat NDA for priority review on Jan. 25. Odevixibat is being evaluated as a treatment option for pruritus in patients with progressive familial intrahepatic cholestasis, a rare inherited progressive liver disease.

Bylvay has been approved as the brand name for odevixibat.

Incyte Hopes Against Hope After Negative Adcom Recommendation

Company: Incyte Corporation INCY
Type of Application: BLA
Candidate: retifanlimab
Indication: Anal Cancer
Date: July 25

Retifanlimab, an intravenous PD-1 inhibitor, is being evaluated as a potential treatment for adult patients with locally advanced or metastatic squamous cell carcinoma of the anal canal, who have progressed on, or who are intolerant of, platinum-based chemotherapy.

Following the acceptance of the NDA on Jan. 21, the PDUFA date was set for July 25.

An FDA panel, which reviewed the application for retifanlimab on Thursday, voted 13-4 that a regulatory decision should be deferred until further data are available from clinical trial POD1UM-303, a confirmatory trial in platinum-naïve advanced SCAC that is currently underway.

‘No or no-go' For Iterum's Antibiotic To Treat Urinary Tract Infection

Company: Iterum Therapeutics plc ITRM
Type of Application: NDA
Candidate: oral sulopenem
Indication: uncomplicated urinary tract infections
Date: July 25

On Jan. 25, the FDA accepted for review the NDA for oral sulopenem as a treatment for uncomplicated urinary tract infections in patients with a quinolone non-susceptible pathogen.

If approved, sulopenem will be the first oral penem in the U.S. and the first new oral treatment for UTIs in over 20 years, the company said.

At the time of the acceptance of the regulatory application, the FDA said it intends to hold an Adcom meeting, without giving a date. Subsequently, in mid-March, the company said in its earnings release the Adcom meeting is scheduled for June 2. In about a month, the FDA said it has decided to postpone the Adcom meeting.

Following a late-cycle meeting with the FDA on May 27, Iterum said the FDA has determined that an Adcom meeting wasn't currently necessary.

Can Ardelyx's Drug To Treat Elevated Blood Phosphate Levels Scrape Through After 3-Month Delay

Company: Ardelyx, Inc. ARDX
Type of Application: NDA
Candidate: tenapanor
Indication: Hyperphosphatemia
Date: July 29

Tenapanor is being evaluated for the control of phosphorus in the blood in adult patients with chronic kidney disease on dialysis. It's estimated to affect more than 745,000 dialysis patients in major developed countries.

Hyperphosphatemia is currently treated with phosphate binders, but without much success. Tenapanor is a non-binder therapy that targets the primary pathway of phosphorus absorption.

The FDA accepted the regulatory application for review on Sept. 15, 2020, and set a PDUFA goal date of April 29. On the D-day, the company communicated FDA's decision to extend the review period by three months. The company suggested following a labeling discussion with the FDA, the agency sought additional information that required the company to submit additional analyses.

The time required for reviewing the additional data was cited as the reason for the extension.

Pfizer Seeks Nod For Atopic Dermatitis Treatment

Company: Pfizer
Type of Application: NDA
Candidate: abrocitinib
Indication: atopic dermatitis
Date: July 27 (estimated)

Abrocitinib is an oral small molecule that selectively inhibits Janus kinase 1, a protein essential for signaling for certain type I and type II cytokines. It is being evaluated for the treatment of atopic dermatitis, a chronic skin disease characterized by inflammation of the skin and skin barrier defects, in patients 12 years and older.

The FDA accepted the NDA for priority review on Oct. 27, 2020, and on April 7, 2021, the company said the review period has been extended by three months to the early third quarter. With the six months applicable to the priority review and the three-month delay, the new PDUFA date is estimated to be July 27.

Adcom Calendar

FDA's Cardiovascular and Renal Drugs Advisory Committee is scheduled to meet July 15 to discuss the NDA for FibroGen, Inc.'s FGEN Roxadustat for the treatment of anemia due to chronic kidney disease in adult patients not on dialysis and on dialysis. FibroGen is partnering with AstraZeneca PLC AZN for Roxadustat. The treatment is already approved in Japan and China.

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