Attention Biotech Investors: Mark Your Calendar For December PDUFA Dates

The U.S. Food and Drug Administration's approval machinery churned out a lot of disappointments in November. Most of the negative verdicts were tied to difficulties the agency has had in inspecting facilities where investigational drugs are being manufactured, amid COVID-19-related restrictions.

Supernus Pharmaceuticals Inc SUPN, Alkermes Plc ALKS, Adamis Pharmaceuticals Corp ADMP, Bristol-Myers Squibb Co BMY, Liquidia Technologies Inc LQDA and Revance Therapeutics Inc RVNC were among the companies that either received complete response letters (which indicate that the application is not ready for approval) or saw the review periods extended.

Notable among the approvals issued during the month were Eiger Biopharmaceuticals Inc's EIGR progeria treatment Zokinvy and Sanofi SA's SNY sutimlimab for treating hemolysis in adult patients with cold agglutinin disease.

Alnylam Pharmaceuticals, Inc. ALNY was fortunate to get the FDA nod for its Oxlumo drug to treat primary hyperoxaluria type 1, ahead of the Dec. 3 PDUFA date.

New molecular entity approvals totaled 44 thus far this year, compared to 41 approvals in the same period last year.

Here are are the key PDUFA dates for December:

Vanda Seeks Twin Nods For Sleep Disorder Drug

Company: Vanda Pharmaceuticals Inc.'s VNDA
Type of Application: NDA & sNDA
Candidate: Hetlioz
Indication: Smith-Magenis Syndrome
Date: Dec. 1

Hetlioz, a melatonin receptor agonist, was approved in Jan. 2014 to treat non—24-hour sleep-wake disorder in totally blind individuals.

Vanda is now seeking an expansion to the label to include Hetlioz capsules. The company has also filed a new drug appication for the liquid formulation of Hetlioz for the treatment of Smith-Magenis Syndrome.

Smith-Magenis Syndrome is a developmental disorder that is caused by a small deletion of human chromosome 17p, or in rare cases, due to a point mutation in the RAI1 gene which resides in the deleted region.

This is estimated to affect one in about 15,200 to 25,000 people in the U.S. The most common symptom is a severe sleep disorder associated with significant disruption in the lives of patients and their families.

Incidentally, Hetlioz faced a rejection as a treatment option for jet lag disorder in mid-2019.

BioCryst's Oral Hereditary Angioedema Drug Awaits Clearance

Company: BioCryst Pharmaceuticals, Inc. BCRX
Type of Application: NDA
Candidate: berotralstat, codenamed BCX7353
Indication: hereditary angioedema, or HAE, attacks
Date: Dec. 3

Berotralstat, which would go by the brand name Orladeyo, is being evaluated as an oral, once-daily treatment for the prevention of HAE attacks. HAE is an inherited condition characterized by recurrent episodes of nonpruritic, nonpitting, subcutaneous or submucosal swelling without the presence of urticarial lesions. It is caused by a low level or improper function of a protein called the C1 inhibitor and affects the blood vessels.

The company expects Orladeyo to fill an important unmet need for an oral HAE prophylactic and generate more than $500 million in global sales, Needham analyst Serge Belanger said in a recent note.

"While we see no significant reason to doubt the FDA approval of Orladeyo by its PDUFA date, we remain skeptical of the product's overall potential given significantly lower efficacy levels (~44% attack rate reduction in ph 3 trials) vs. current injectable therapies with 80%-90% efficacy," the analyst said.

Will MacroGenics' Breast Cancer Drug Clear The FDA Hurdle?

Company: MacroGenics Inc MGNX & Zai Lab Ltd – ADR ZLAB
Type of Application: BLA
Candidate: margetuximab
Indication: Breast cancer
Date: Dec. 18

Margetuximab, a Fc-engineered, anti-HER2 mAb, is being evaluated in combination with chemotherapy for treating patients with with metastatic HER2-positive breast cancer.

MacroGenics has partnered with Zai Lab in Greater China for the indication.

Amgen's Rituxan Biosimilar Faces FDA Decision

Company: Amgen, Inc. AMGN/ AbbVie Inc ABBV
Type of Application: BLA
Candidate: ABP 798
Indication: Cancer
Date: Dec. 19

ABP 798 is a biosimilar candidate to Roche Holdings AG's Basel ADR Common Stock RHHBY Rituxan.

Rituxan is an anti-CD20 monoclonal antibody, which has been approved for many indications, including non-Hodgkin's lymphoma, chronic lymphocytic leukemia, granulomatosis with polyangiitis and microscopic polyangiitis with glucocorticoids.

The active ingredient of ABP 798 is a monoclonal antibody with the same amino acid sequence as Rituxan.

Related Link: Biotech Stocks With Material Catalysts In Q4

Can Fibrogen/AstraZeneca's CKD-related Anemia Drug Win The Nod?

Company: FibroGen Inc FGEN & AstraZeneca plc AZN
Type of Application: NDA
Candidate: roxadustat
Indication: anemia of chronic kidney disease
Date: Dec. 20

Roxadustat is an orally administered, small molecule HIF-PH inhibitor that promotes erythropoiesis, which is the process of producing red blood cells or erythrocytes.

The NDA seeks approval of roxadustat for the treatment of anemia in chronic kidney disease, in both non-dialysis-dependent and dialysis-dependent patients.

Fibrogen is collaborating with AstraZeneca on the development and commercialization of roxadustat for the treatment of anemia in the U.S., China and other markets in the Americas and in Australia, New Zealand and Southeast Asia.

Myovant's Binary Event For Relugolix In Prostate Cancer In The Cards

Company: Myovant Sciences Ltd MYOV
Type of Application: NDA
Candidate: relugolix
Indication: prostate cancer
Date: Dec. 20

In late June, the FDA accepted for priority review the NDA for once-daily, oral relugolix, 120 mg, for the treatment of men with advanced prostate cancer.

Relugolix is an oral gonadotropin-releasing hormone receptor antagonist that reduces production of testicular testosterone, a hormone known to stimulate the growth of prostate cancer, and ovarian estradiol, a hormone known to stimulate the growth of uterine fibroids and endometriosis.

Urovant Seeks Approval For Drug to Treat Overreactive Bladder

Company: Urovant Sciences Ltd UROV
Type of Application: NDA
Candidate: vibegron
Indication: overactive bladder
Date: Dec. 26

The FDA accepted the NDA for a review March 5, with the regulatory filing seeking approval for once-daily 75 mg vibegron for the treatment of patients with overactive bladder with symptoms of urge urinary incontinence, urgency and urinary frequency.

‘Go or No-go' For Viatris' Copycat Version of Roche's Top-selling Cancer Drug

Company: Viatris Inc VTRS
Type of Application: BLA
Candidate: MYL-1402O
Indication: Multiple cancer types
Date: Dec. 27

Viatris, which formed following the merger of the erstwhile generic pharma company Mylan and Pfizer Inc.'s PFE generic unit Upjohn, has a tryst with the FDA in late December. The regulatory agency is scheduled to rule on the BLA for MYL-1402O, a proposed alternative to Roche's Avastin, for review under the 351(k) pathway. This is being developed in partnership with India's Biocon.

The BLA seeks approval of MYL-1402O for first-line and second-line treatment of patients with metastatic colorectal cancer in combination with fluorouracil-based chemotherapy; first-line use for patients with non-squamous non-small cell lung cancer; recurrent glioblastoma; metastatic renal cell carcinoma in combination with interferon alfa; and persistent, recurrent or metastatic cervical cancer.

Can Second Time Be Charm For Osmotica

Company: Osmotica Pharmaceuticals PLC OSMT
Type of Application: NDA
Candidate: Ontinua (arbaclofen) ER
Indication: spasticity in patients with MS
Date: Dec. 29

Osmotica originally submitted the NDA for arbaclofen ER in 2015, with the FDA issuing a complete response in June 2016, citing irregularities and deviations from good clinical practices at study sites of the initial Phase 3 trial.

The company submitted in late June an amended NDA, and in late July, it said the regulatory agency had deemed its submission as a complete, class 2 response.

"Management noted that the questions received from FDA so far have been reasonable and confirmatory in nature, and that it feels very good about its answers to the agency's information requests," SVB Leerink analyst Ami Fadia said in a recent note.

"All in all, it clearly sees great potential in arbaclofen, but is cautiously optimistic on the approval given the history of the product."

Scpharma Hopes For No Heartache In Second Try For Heart Failure Drug

Company: Scpharmaceuticals Inc SCPH
Type of Application: NDA
Candidate: Furoscix
Indication: worsening heart failure due to congestion
Date: Dec. 30

Furoscix is a proprietary, subcutaneously-delivered furosemide solution, as an outpatient alternative for the treatment of worsening congestive heart failure.

This is scPharma's second try for Furoscix following a CRL issued in 2018, wherein the agency sought additional human factor studies, device modifications and potentially a clinical validation study.

The PDUFA event is a significant catalyst for the stock, SVB Leerink analyst Fadia said.

The analyst said she is cautiously optimistic on the approval, given the regulatory history, and put an 80% probability of success on approval. If approved, the analyst models a price target for $20 for the shares.

Athenex Gears to Face Verdict On Drug to Treat Precancerous Skin Condition

Company: Athenex Inc ATNX
Type of Application: NDA
Candidate: tirbanibulin ointment
Indication: actinic keratosis
Date: Dec. 30

The NDA for tirbanibulin ointment was accepted for review March 9. Athenex has granted Almirall, S.A., an exclusive license for R&D and commercializing tirbanibulin ointment in the U.S. and European countries, including Russia.

Actinic Keratosis is the most common pre-cancerous condition in dermatology and affects more than 55 million Americans, and accounts for between 14-29% of dermatologist visits in the U.S., Athenex said.

Vertex Seeks Label Expansions For Cystic Fibrosis Medications

Company: Vertex Pharmaceuticals Incorporated VRTX
Type of Application: sNDAs
Candidate: Trikafta (elexacaftor, tezacaftor and ivacaftor), Symdeko (tezacaftor and ivacaftor) and Kalydeco (ivacaftor)
Indication: cystic fibrosis
Date: Dec. 30

The sNDAs were accepted by the FDA for review Sept. 1. These regulatory submissions are intended to expand the labels for Trikafta, Symdeko and Kalydeco to include additional rare CFTR mutations, Vertex said. Potential approvals will also allow certain people with cystic fibrosis, who are currently eligible for Kalydeco to become eligible for Symdeko or Trikafta and certain people currently eligible for Symdeko tp become eligible for Trikafta, it added.

Adcom Calendar

The Center for Biologics Evaluation and Research's Vaccines and Related Biological Products Advisory Committee, or VRBPAC, will meet Dec. 10, in an open session to discuss emergency use authorization of the vaccine candidate developed by Pfizer-BioNTech SE – ADR BNTX for the prevention of COVID-19 in individuals 16 years of age and older.

The companies filed with the FDA an application for EUA Nov. 20.

Moderna MRNA is also likely to meet with VRBPAC once it mandated safety data is accrued and it files for EUA.

Related Link: AstraZeneca Analyst Flags Lack Of Details In Interim COVID-19 Vaccine Data

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