Attention Biotech Investors: Mark Your Calendar For These July PDUFA Dates

Zinger Key Points
  • PDUFA goal date is a make-or-break catalyst for a biopharma
  • It's the date by which FDA is required to announce its verdict on a regulatory application
  • The unfolding week's PDUFA catalyst is fairly light

The biotech sector is poised to end June on a positive note, despite largely disappointing regulatory decisions scheduled during the month, thanks to a reversal in broader market sentiment.

Only one new molecular entity (NME) has been approved thus far in June. NMEs are a key measure of drug innovation as they contain active ingredients yet to be approved by the Food and Drug Administration (FDA). Alnylam Pharmaceutical Inc's ALNY RNAi therapeutic Vutrisiran received approval for treating polyneuropathy of hereditary transthyretin-mediated amyloidosis in adults.

Acer Therapeutics Inc ACER and partner Relief Therapeutics Holding AG RLFTF received a  complete response letter for their urea cycle disorder drug

Bristol-Myers Squibb Co BMY received approval for its supplemental biologics license application (sBLA) for Breyanzi for large B-cell lymphoma, while it withdrew its application for Reblozyl as a treatment option for anemia in adults with non-transfusion dependent beta-thalassemia.

Spero Therapeutics Inc's SPRO new drug application (NDA) for its complicated urinary-tract infection candidate was shot down by the FDA with a CRL.

On the other hand, Regeneron Pharmaceuticals Inc. REGN and Sanofi SNY received approval for expanding the label of Dupixent to treat atopic dermatitis in children. Rhythm Pharmaceuticals, Inc.'s RYTM rare, genetic obesity and hunger treatment received FDA approval.

Related Link:  Merck/Seagen Potential Deal Talks Pick Up Speed: WSJ 

Here are the key regulatory decisions scheduled for July:


Horizon Therapeutics Seeks Label Expansion For Gout Treatment

Company:  Horizon Therapeutics Ltd. HZNP

Type of Application:  sBLA

Candidate:  Krystexxa

Indication:  gout

Date:  July 7

Krystexxa, developed by Savient, received its first approval in 2010 for treating gout in adult patients Savient was subsequently bought by Crealta Holdings, which in turn was bought by Horizon in 2015.

Horizon is now seeking to expand the label of Krystexxa, which would allow the company's commercial team to promote the drug, plus methotrexate, to physicians for gout. The application has a priority review status.

Gout is a more commonly found and complex form of arthritis that results from the crystallization and deposition of uric acid in the joints.

In 2021, Krystexxa fetched the company $565.5 million in sales, representing a 39% year-over-year growth.


Can BeiGene Snag First Overseas Approval For Anti-PD-1 Antibody?


Company:  BeiGene Ltd. BGNE

Type of Application:  BLA

Candidate:  tislelizumab

Indication:  esophageal squamous cell carcinoma

Date: July 12

Tisleizumab is China-based biopharma BeiGene's anti-PD-1 antibody, and the drug has received approvals for five indications in China. The company filed its first excluding-China application with the U.S. FDA for treating patients with unresectable, recurrent, locally advanced esophageal squamous cell carcinoma after prior system therapy.

Tisleizumab is also under regulatory review in China for the same indication.

Esophageal cancer is the most common malignant tumor in the digestive tract, with over 18,400 new cases diagnosed each year in the U.S., according to statistics shared by the company.


Regeneron Awaits Full Approval For COVID-19 Antibody Cocktail


Company:  Regeneron

Type of Application:  BLA

Candidate:  REGEN-COV

Indication:  COVID-19

Date:  July 13

REGEN-COV is an antibody cocktail, comprising casirivimab and imdevimab, and Regeneron is seeking full approval for using it in hospitalized COVID-19 patients and for prophylactic treatment in some individuals.

The earlier Prescription Drug User Fee Act (PDUFA) date was extended by three months to July 13, as the regulator required more time to review the additional data Regeneron submitted to support pre-exposure prophylactic use.

REGEN-COV has been available to U.S. patients since November 2020 under emergency use authorization (EUA). The EUA was amended in January 2022 to exclude its use in geographic regions, where infection or exposure is likely due to a variant that is not susceptible to the treatment.


Go Or No-go For Incyte's Opzelura In Expanded Indication?


Company: Incyte Corporation INCY

Type of Application:  sNDA

Candidate:  Opzelura (ruxolitinib cream)

Indication:  vitiligo

Date:  July 18

Opzelura is a novel cream formulation of Incyte's selective JAK1/JAK2 inhibitor ruxolitinib. It is the first and only topical JAK inhibitor approved for use in the U.S. for the topical short-term and non-continuous chronic treatment of mild to moderate atopic dermatitis in non-immunocompromised patients 12 years of age and older.

The company is now seeking approval of the same for vitiligo, an autoimmune disorder. The skin loses its pigment cells in a person afflicted with vitiligo, which causes discolored patches.


Will Arcutis Get Its Maiden Approval?


Company: Arcutis Biotherapeutics, Inc. ARQT

Type of Application:  NDA

Candidate:  roflumilast cream

Indication:  plaque psoriasis

Date:  July 29

Roflumilast cream, according to Arcutis, is a once-daily cream formulation being developed for plaque psoriasis and atopic dermatitis. The application for clearing the investigational treatment for plaque psoriasis was accepted for review in December 2021, with a PDUFA goal date of July 29, 2022.

The company said in an investor presentation in March that about 9 million people suffer from plaque psoriasis in the U.S. alone, with 90% of them treated with topical drugs. Existing topical therapies have several shortcomings related to safety and tolerability.

Arcutis' roflumilast could be the first topical cream formulation to be approved in the U.S. for plaque psoriasis.

Related Link: US Expands Monkeypox Testing Capacity To Five Commercial Lab Companies

Photo via grandbrothers on Shutterstock


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