Attention Biotech Investors: Mark Your Calendar For September PDUFA Dates

August proved to be a mixed month for regulatory approvals, with the Food and Drug Administration approving a few, turning down a few others and delaying the remaining.

New molecular entity approvals – a measure of innovation in the biopharma sector – numbered four for the month, taking the total for the year to 36, two less than relative to the same period in 2020. NMEs are new drugs containing an active ingredient that hasn't yet been marketed in the U.S.

Sanofi SNY's Nexviazyme, an enzyme replacement therapy for Pome disease, Cara Therapeutics, Inc.'s CARA Korsuva as a treatment option for moderate-to-severe pruritus in hemodialysis patients and Ascendis Pharma A/S ASND's pediatric growth hormone deficiency treatment Skytrofa were among the NMEs approved during the week.

Jazz Pharmaceuticals plc JAZZ succeeded in getting a label expansion for its sleep disorder drug. On the other hand, Sesen Bio, Inc.'s SESN bladder cancer drug vicineum was issued a complete response letter.

Here's a look at what's ahead in September.

Check out Benzinga's full FDA calendar!

Impel NeuroPharma Aiming to Transform to a Commercial-stage Biopharma With its Migraine Drug Approval

• Company: Impel NeuroPharma, Inc. IMPL
• Type of Application: NDA
• Candidate: Trudhesa (INP104)
• Indication: acute migraine
• Date: Sept. 6

Impel's lead candidate INP104 is dihydroergotamine mesylate delivered directly into the vascular-rich upper nasal space using its proprietary Precision Olfactory Delivery technology. It's being investigated as a treatment option for migraine headaches, with or without aura in adults.

The FDA accepted the application for a standard review Jan. 20.

"We believe there will be eventual significant adoption for Trudhesa, as it's set to become another needed option following triptan failures (for which 40-50% of patients are not well-served) and before – or after – treatment with newer oral CGRPs," Cowen analyst Ken Cacciatore said in a note.

The analyst assumes a fourth-quarter launch, with U.S. sales estimated to easily exceed $500 million, or roughly 5% of the acute market, within five years.

Calliditas Awaits Word On Its Kidney Inflammation Drug

• Company: Calliditas Therapeutics AB CALT
• Type of Application: NDA
• Candidate: Nefecon
• Indication: IgA nephropathy
• Date: Sept. 15

The company filed the application on March 15, and the FDA accepted it for priority review on April 28. Nefecon is a patented oral formulation of budesonide – for targeted release. The formulation is designed to deliver the drug to the Peyer's patch region of the lower small intestine, where the disease originates, as per the predominant pathogenesis models.

Nefecon is derived from the TARGIT technology, which allows for the substance to pass through the stomach and intestine without being absorbed, and to be released in a pulse-like fashion only when it reaches the lower small intestine.

IgA nephropathy, also known as Berger's disease, is a kidney disease that occurs when IgA deposits build up in the kidneys, causing inflammation that damages kidney tissues. IgA is an antibody made by the immune system to protect the body from foreign substances such as bacteria or viruses.

"Based on the successful Ph2 and Ph3 trial results, we remain positive and optimistic about Nefecon's FDA approval," HC Wainwright analyst Edwin Zhang said in a recent note.

Go or no-go For Samsung Bioepis' Lucentis Biosimilar

• Company: Biogen Inc. BIIB and Samsung Bioepis
• Type of Application: Biologic license application
• Candidate: SB11
• Indication: a biosimilar to Roche Holding AG RHHBY indicated for a slew of retinal vascular disorders
• Date: Sept. 17 (estimated based on 10-month standard review from the date of filing)

Lucentis is an anti-VEGF therapy for retinal vascular disorders, which are a leading cause of blindness in the U.S. and SB11 is a proposed biosimilar to the therapy. Samsung Bioepis has a commercialization agreement with Biogen for SB11 in the U.S.

BeiGene Seeks Label Expansion For Blood Cancer Drug

• Company: BeiGene, Ltd. BGNE
• Type of Application: supplemental NDA
• Candidate: Brukinsa
• Indication: marginal zone lymphoma
• Date: Sept. 19

Brukinsa is a small molecule inhibitor of Bruton's tyrosine kinase discovered by BeiGene scientists that's currently being evaluated globally in a broad clinical program as a monotherapy and in combination with other therapies to treat various B-cell malignancies.

The FDA has now accepted the regulatory application for priority review as a treatment option for adult patients with marginal zone lymphoma, who have received at least one prior anti-CD20-based therapy and granted priority review.

Marginal zone lymphoma is a serious disease diagnosed in more than 2,000 patients every year in the U.S., with no clear standard of care.

Will Incyte's Twin Binary Events Fructify After 3-Month Delays

• Company: Incyte Corporation INCY
• Type of Application: NDA/sNDA
• Candidate: ruxolitinib cream/ ruxolitinib (Jakafi)
• b> Indication: atopic dermatitis/ steroid-refractory chronic graft-versus-host disease
• Date: Sept. 21

Ruxolitinib cream, a selective JAK1/JAK2 inhibitor designed for topical application, is an investigational treatment for atopic dermatitis, a type of eczema. Incyte's NDA was accepted for priority review on Feb. 19. Since the company submitted a priority review voucher along with the NDA application for ruxolitinib cream, the review period was shortened by four months.

In mid-June, the company communicated a three-month extension in the review period to allow the agency time to review additional analyses of previously submitted data in response to the FDA's information request.

The review period for the regulatory application for Jakafi as a treatment option for steroid-refractory chronic graft-versus-host disease in adult and pediatric patients 12 years and older, was also extended by three months, rendering the new PDUFA date at Sept. 12.

Verrica Hopes to Put Behind Disappointments With Viral Skin Infection Drug Approval

• Company: Verrica Pharmaceuticals Inc. VRCA
• Type of Application: NDA
• Candidate: VP-102
• Indication: treatment of molluscum contagiosum

VP-102, or cantharidin 0.7% topical solution, is being evaluated as a potential treatment option for molluscum contagiosum and common warts. Molluscum, according to Verrica, is a highly contagious viral skin infection affecting approximately six million people, primarily children, in the U.S.

Verrica submitted the NDA on Sept. 13, 2019, with the FDA accepting the application for review on Nov. 26. FDA handed down a complete response letter later, and the company resubmitted the application on Dec. 23, 2020. The PDUFA goal date for the review of the resubmitted application was set for June 23, 2021.

Mirum Expects Smooth Sailing For Drug to Treat Itching Associated with Rare Liver Disease

• Company: Mirum Pharmaceuticals, Inc. MIRM
• Type of Application: NDA
• Candidate: maralixibat
• Indication: cholestatic pruritus in patients with Alagille syndrome
• Date: Sept. 29

Maralixibat, an oral apical sodium dependent bile acid transporter inhibitor, is being investigated as a treatment option for cholestatic pruritus in patients with Alagille syndrome, one year of age and older. ALGS is a rare liver disease for which there are currently no approved therapies.

The FDA has accepted the regulatory application for priority review on May 29.

AbbVie Awaits Approval For Migraine Preventive Treatment

• Company: AbbVie Inc. ABBV
• Type of Application: NDA
• Candidate: atogepant
• Indication: preventive treatment of migraine
• Date: Late third quarter

Atogepant, an investigational orally administered calcitonin gene-related peptide receptor antagonist, is being evaluated for the preventive treatment of migraine in adults who meet the criteria for episodic migraine.

Episodic migraines are a debilitating neurological disease affecting 39 million people in the U.S., the company said.

Can Alvotech Secure Nod For Humira Biosimilar?

• Company: Alvotech
• Type of Application: BLA
• Candidate: AVT02
• Indication: proposed biosimilar for AbbVie's Humira
• Date: September

Alvotech, an Iceland-based biotech, through its U.S. subsidiary has filed for the approval of AVT02, a monoclonal antibody and a proposed biosimilar to Humira. AVT02 is one of five product candidates from Alvotech that will be commercialized, upon approval, by Teva Pharmaceutical Industries Limited TEVA in the U.S. following an exclusive 2019 strategic partnership.

Humira is AbbVie's top-selling immunology drug that is approved for a slew of autoimmune and inflammatory diseases. It fetched the company $5.07 billion in sales in the second quarter.