Attention Biotech Investors: Mark Your Calendar For June PDUFA Dates

The FDA review machinery worked overtime in May, completing reviews of several drug applications. Except for stray rejections or delays, the agency churned out mostly positive outcomes.

See also: Best Biotech Stocks Right Now

New molecular entity approvals totaled five during the month. NMEs are drugs with active moieties or ingredients that have never been approved by the FDA previously, and are therefore indicators of drug innovation.

NME approvals for the month, included Apellis Pharmaceuticals, Inc.'s APLS Empaveli, indicated for the treatment of paroxysmal nocturnal hemoglobinuria, Johnson & Johnson's JNJ Rybrevant for a subtype of non-small cell lung cancer, Lantheus Holdings, Inc.'s LNTH Pylarify, a fluorinated PSMA-targeted PET imaging agent for detecting prostate cancer, Amgen, Inc.'s KRAS G12C-targeted lung cancer treatment Lumakras and BridgeBio Pharma, Inc.'s BBIO Truseltiq, indicated for biliary tract cancer.

Meanwhile, Eton Pharmaceuticals, Inc.'s ETON new drug application for dehydrated alcohol injection was slapped with a complete response letter.

Here are the key FDA approvals scheduled for June.

SCYNEXIS Awaits Nod For Anti-fungal Treatment For Vaginal Yeast Infection

  • Company: SCYNEXIS, Inc. SCYX
  • Type of Application: NDA
  • Candidate: SCY-078 (oral ibrexafungerp)
  • Indication: vulvovaginal candidiasis
  • Date: June 1

The FDA accepted the application for priority review on Dec. 7. The investigational drug has both Qualified Infectious Disease Product, or QIDP, and Fast Track designations. The QIDP status vests the company with five years of market exclusivity, in addition to the five years of exclusivity as a chemical entity.

The company indicated it's continuing preparations for a commercial launch in the U.S. in the second half of 2021.

Vulvovaginal candidiasis, commonly known as a vaginal yeast infection due to Candida, is the second most common cause of vaginitis. The currently available treatment option is fluconazole approved way back in 1990. Fluxconzole faces shortcomings such as increasing resistance to it and upping the risk of miscarriage.

H.C. Wainwright estimates peak sales potential of $170 million for ibrexafungerp, assuming conservative net pricing of $170 million per prescription at launch and a conservative peak 5% market share.

Can Liminal's Plasminogen Replacement Therapy Cross The FDA Hurdle After Several Hiccups?

  • Company: Liminal BioSciences Inc. LMNL
  • Type of Application: biologic license application
  • Candidate: Ryplazim (plasminogen)
  • Indication: Congenital plasminogen deficiency
  • Date: June 5

Plasminogen, which will go by the trade name Ryplazim, is being evaluated for the treatment of congenital plasminogen deficiency. Plasminogen is a naturally occurring protein that is synthesized by the liver and circulates in the blood, and it is the main enzyme involved in the lysis of blood clots and clearance of extravasated fibrin.

Plasminogen, therefore, is vital for wound healing, cell migration, tissue remodeling, angiogenesis and embryogenesis.

The original BLA filed by Prometic Life Sciences in 2017 received a complete response the same year. A resubmission was done by Prometic, a subsidiary of Liminal, last September and following the acceptance of the filing, the FDA set a decision date of March 7. In early November, Liminal communication a three-month extension announced by the FDA, pushing the decision date to June 7.

‘No or no-go' For Biogen's Controversial Alzheimer's Drug

  • Company: Biogen Inc. BIIB
  • Type of Application: BLA
  • Candidate: Aducanumab
  • Indication: Alzheimer's disease
  • Date: June 7

Biogen resurrected aducanumab after shelving a couple of late-stage studies, thanks to subset analysis of data. It's an investigational human monoclonal antibody. The company had licensed the candidate from Neurimmune and is developing it along with Eisai Co., Ltd. ESALY.

Biogen's BLA was accepted for review in August 2020, with a PDUFA date of March 8. An Adcom panel, which reviewed the BLA in November, voted against approving it. The negative verdict did not douse hopes of an eventual approval, given the dire need for a drug against this neurological disorder.

The company communicated in January a three-month extension in review period to June 7.

Related Link: Why This Pfizer Analyst Is Sidelined After Beat-And-Raise Q1

Alexion On Track For Third Label Expansion of Blood Disorder Drug

  • Company: Alexion Pharmaceuticals, Inc. ALXN
  • Type of Application: supplemental NDA
  • Candidate: Ultomiris
  • Indication: paroxysmal nocturnal hemoglobinuria in children and adolescents
  • Date: June 7

Alexion's Ultomiris was initially approved for PNH in adults in December 2018. Since then, it has received two label expansions. Alexion, which has agreed to be acquired by AstraZeneca Plc AZN, is looking to expand the label to include the indication of PNH in children and adolescents.

PNH is a blood disorder characterized by complement-mediated destruction of the red blood cells that can cause a wide range of debilitating symptoms and complications, including thrombosis, which can occur throughout the body, and result in organ damage and premature death.

Can Vertex's Triple Combo Cystic Fibrosis Drug Get The Nod For Pediatric Usage

  • Company: Vertex Pharmaceuticals Incorporated VRTX
  • Type of Application: sNDA
  • Candidate: Trikafta
  • Indication: cystic fibrosis in children
  • Date: June 8

Trikafta, a triple combination of elexacaftor/tezacaftor/ivacaftor and ivacaftor, received its first approval in October 2019 as a treatment option for cystic fibrosis in people, ages 12 years and older, who have at least one F508del mutation in the cystic fibrosis transmembrane conductance regulator gene.

In December, the FDA expanded the eligibility for Trikafta to include people with cystic fibrosis, ages 12 years and older with certain mutations in the cystic fibrosis transmembrane conductance regulator.

Pfizer's Pneumonia Vaccine Targeting 20 Strains of Bacteria Awaits FDA Nod

  • Company: Pfizer Inc. PFE
  • Type of Application: BLA
  • Candidate: 20-valent pneumococcal conjugate (20vPnC) vaccine
  • Indication: Pneumonia
  • Date: June 8 (estimated)

The 20vPnC candidate vaccine is in development for the prevention of invasive disease and pneumonia caused by 20 serotypes of Streptococcus pneumoniae in the vaccine in adults ages 18 years and older.

Pfizer announced FDA's acceptance for priority review its BLA for the vaccine candidate on Dec. 8, 2020, with a PDUFA date of June.

Takeda Seeks Approval For Drug to Treat Inflammation of Esophagus

  • Company: Takeda Pharmaceutical Company Limited TAK
  • Type of Application: NDA
  • Candidate: TAK-721
  • Indication: eosinophilic esophagitis
  • Date: June 15 (estimated)

Eosinophilic esophagitis is a chronic, immune-mediated, inflammatory disease localized in the esophagus, and it currently doesn't have an FDA-approved treatment. TAK-721 is a mucoadherent topically active oral viscous formulation of budesonide.

The regulator accepted for priority review Takeda's application on Dec. 15, 2020.

Blueprint's Ayvakit Up Before FDA For Label Expansion

  • Company: Blueprint Medicines Corporation BPMC
  • Type of Application: sNDA
  • Candidate: Ayvakit
  • Indication: advanced systemic mastocytosis
  • Date: June 16

Ayvakit was initially approved by the FDA in January 2020 for the treatment of adults with unresectable or metastatic gastrointestinal stromal tumor harboring a PDGFRA exon 18 mutation.

The company is now looking to expand the label of the treatment to include advanced systemic mastocytosis, which is a group of rare diseases in which uncontrolled growth and accumulation of a type of white blood cells called mast cells occurs in one or more organs, including the gastrointestinal tract, spleen, lymph nodes and bone marrow.

Can Orphazyme Leave The ALS Debacle Behind?

  • Company: Orphazyme A/S ORPH and CytRx Corporation CYTR
  • Type of Application: NDA
  • Candidate: Arimocolmol
  • Indication: Niemann-Pick Disease Type C
  • Date: June 17

NPC, according to the company, is a rare, relentlessly progressive, neurodegenerative disease, with an estimated incidence of one in 100,000 live births. The FDA had accepted the NDA for priority review on Sept. 16, 2020, and set a PDUFA goal date of March 17.

In Dec. 2020, the company said the FDA has delayed the decision by three months to June 17.

Orphazyme, a Denmark-based biopharma, is collaborating with Cytrx Corp. in developing arimocolmol, the only drug candidate in its pipeline. Incidentally, the drug failed in clinical trials for amyotrophic lateral sclerosis.

Will Eton's Ready-to-use Low Blood Pressure Medication Cross FDA Hurdle?

  • Company: Eton Pharma
  • Type of Application:
  • Candidate: Ready-to-Use ephedrine injection
  • Indication: low blood pressure
  • Date: June 18

Eton sees market opportunity for the RTU ephedrine injection to be more than 10 million RTU units annually. This product candidate is an innovative ready-to-use formulation of a molecule that is currently approved in a concentrated formulation that must be diluted prior to administration to patients.

Eton's development partner, Sintetica, has submitted the product's NDA.

Incyte has Twin Binary Events In June

  • Company: Incyte Corporation INCY
  • Type of Application: NDA/ sNDA
  • Candidate: ruxolitinib cream/ ruxolitinib (Jakafi)
  • Indication: atopic dermatitis/ steroid-refractory chronic graft-versus-host disease
  • Date: June 21/22

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

The company is also seeking label expansion for Jakafi as a treatment option for steroid-refractory chronic graft-versus-host disease in adult and pediatric patients 12 years and older.

Ascendis Confident of Pediatric Growth Hormone Deficiency Prodrug Approval

  • Company: Ascendis Pharma A/S ASND
  • Type of Application: BLA
  • Candidate: TransCon hGH
  • Indication: Pediatric growth hormone deficiency
  • Date: June 25

TransCon hGH is being evaluated for the treatment of pediatric patients with growth hormone deficiency. It is an investigational prodrug of human growth hormome somatropin. The company is also developing a proprietary auto-injector to administer TransCon hGH, stable at room temperature, in dual-chamber cartridges.

Ascendis said in its first-quarter earnings report that pre-launch commercial activities are ongoing and the treatment candidate is scheduled to be launched in the U.S. in the third quarter of 2021.

"All FDA information requests relating to our BLA for TransCon hGH for the treatment of pediatric GHD have been responded to with no questions currently outstanding," it had said.

Decision Due On MediWound's Biologic Treatment For Thermal Wounds

  • Company: MediWound Ltd. MDWD & Vericel Corporation VCEL
  • Type of Application: BLA
  • Candidate: NexoBrid
  • Indication: severe thermal burns
  • Date: June 29

The NexoBrid BLA, which requests approval for eschar removal in adults with deep partial-thickness and/or full-thickness thermal burns, was accepted for review in mid-September 2020. The FDA assigned a PDUFA goal date of June 29.

NexoBrid, a concentrate of proteolytic enzymes enriched in Bromelain, is a topically administered biological product that enzymatically removes non-viable burn tissue, or eschar, in patients with deep partial and full-thickness thermal burns within four hours of application without harming viable tissue.

NexoBrid is approved in the European Union and other international markets.

MediWound, an Israeli biopharma, has granted the North American license to NexoBrid to Vericel under a May 2019 agreement.

AbbVie's Rheumatoid Arthritis Drug Prepping For Label Expansion

  • Company: AbbVie Inc. ABBV
  • Type of Application: sNDA
  • Candidate: Rinvoq (updacitinib)
  • Indication: active psoriatic arthritis.
  • Date: late second quarter.

AbbVie announced that the FDA extended the review period for sNDA for Rinvoq in the treatment of adult patients with active psoriatic arthritis to late second quarter. The company clarified that the FDA made an information request for an updated assessment of the benefit-risk profile. Following the submission of information by AbbVie, the FDA required additional time for a full review of the submission.

Adcom Meetings

FDA's Vaccines and Related Biological Products Advisory Committee is scheduled to meet June 10 to discuss, in general, data needed to support authorization and/or licensure of COVID-19 vaccines for use in pediatric populations.

The Oncologic Drug Advisory Committee will meet June 24 to discuss Incyte's BLA for retifanlimab injection for the proposed indication of treating adult patients with locally advanced or metastatic squamous carcinoma of the anal canal, who have progressed on or who are intolerant of platinum-based chemotherapy.

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