Attention Biotech Investors: Mark Your Calendar For May PDUFA Dates

Following a mixed month in April, the Food and Drug Administration sets out to review another slate of drugs in May.

ACADIA Pharmaceuticals Inc.'s ACAD Nuplazid failed to get label expansion to include the indication of dementia-related psychosis. The FDA did not keep its tryst with the decision dates for ACADIA Pharmaceuticals Inc.'s ACAD pain drug and Sol-Gel Technologies Ltd.'s SLGL investigational skin inflammation treatment.

Protalix BioTherapeutics, Inc.'s PLX Fabry disease treatment candidate also faced rejection at the FDA altar. The regulatory agency extended the review period for Pfizer Inc.'s PFE atopic dermatitis drug.

On the other hand, Supernus Pharmaceuticals, Inc.'s SUPN attention deficit hyperactivity drug cleared the FDA hurdle in its second attempt. And GlaxoSmithKline plc's GSK Jemperli, a new molecular entity, won the nod for advanced endometrial cancer.

Another the NMEs approved during the month was ADC Therapeutics SA's ADCT Zynlonta in relapsed or refractory large B-cell lymphoma.

Here are the key FDA approvals scheduled for May:

Will Third Time Be Charm For Heron?

  • Company: Heron Therapeutics, Inc. HRTX
  • Type of Application: NDA
  • Candidate: HTX001
  • Indication: postoperative pain
  • Date: May 12

Heron's HTX-011, a non-opioid pain drug, is a combo drug consisting of bupivacaine and a low dose of non-steroidal anti-inflammatory drug meloxicam.

The NDA was originally submitted in October 2018, and in response to the application, the FDA issued a complete response letter in April 2019, citing the need for additional CMC and non-clinical information.

Heron resubmitted the NDA in October 2019, and in Feb. 2020, the company said the FDA extended the review period by three months, rendering the PDUFA data on June 26. A second complete response letter was handed out on June 29, with the FDA seeking additional non-clinical information.

The company went on to file a resubmission on Nov. 12. If approved, HTX-011 will compete with Pacira Biosciences Inc's PCRX Exparel.

Apellis Awaits Nod For Blood Disorder Drug

  • Company: Apellis Pharmaceuticals, Inc. APLS
  • Type of Application: NDA
  • Candidate: pegcetacoplan
  • Indication: paroxysmal nocturnal hemoglobinuria (PNH)
  • Date: May 14

Pegcetacoplan is an investigational, targeted C3 therapy designed to regulate excessive activation of the complement cascade, a part of the body's immune system. The company is evaluating pegcetacoplan in several clinical studies across hematology, ophthalmology, nephrology and neurology.

PNH is a rare, chronic and life-threatening blood disorder characterized by the destruction of oxygen-carrying red blood cells through extravascular and intravascular hemolysis, or rupturing of red blood cells.

The FDA accepted the application for priority review on Nov. 16 and assigned a PDUFA goal date of May 14.

Can Sanofi's Enzyme Replacement Therapy For Pompe Disease Cross The FDA Hurdle?

  • Company: Sanofi SNY
  • Type of Application: biologic license application
  • Candidate: avalglucosidase alfa
  • Indication: Pompe disease
  • Date: May 18

Avalglucosidase alfa, an enzyme replacement therapy, is being evaluated for the treatment of patients with Pompe disease, a rare degenerative muscle disorder that can impact an individual's ability to move and breathe. It affects an estimated 3,500 people in the U.S. and can manifest at any age from infancy to late adulthood.

Bristol-Myers Squibb Seeks Label Expansion For Cancer Immunotherapy Opdivo

  • Company: Bristol-Myers Squibb Company BMY
  • Type of Application: sBLA
  • Candidate: Opdivo
  • Indication: esophageal cancer
  • Date: May 20

The pharma giant is seeking label expansion for Opdivo as a treatment option for patients with surgically removed esophageal or gastroesophageal junction cancer under certain circumstances.

The regulatory application was filed based on results from the CheckMate -577 trial. The FDA accepted the application for priority review on Jan. 20, assigning it a PDUFA goal date of May 20.

Related Link: Why This Bristol-Myers Squibb Stock Analyst Is Turning Bullish On The Pharma Company's Pipeline

No Or No-Go For Eton's Dehydrated Alcohol Injection

  • Company: Eton Pharmaceuticals, Inc. ETON
  • Type of Application: Dehydrated alcohol injection
  • Candidate: NDA
  • Indication: methanol poisoning
  • Date: May 27

The regulatory application for dehydrated alcohol injection was accepted for review in October 2020. Eton's application has an orphan drug designation for the treatment of methanol poisoning and is expected to receive seven years of market exclusivity upon its approval.

The U.S. market for dehydrated alcohol injection, according to Eton, is estimated to be greater than $100 million annually and the drug is currently only supplied by a single manufacturer.

Decision Due On Lantheus' Prostate Cancer Imaging Agent

  • Company: Lantheus Holdings, Inc. LNTH
  • Type of Application: NDA
  • Candidate: PyL
  • Indication: prostate cancer
  • Date: May 28

The FDA accepted the NDA for priority review in December. PyL is an investigational fluorinated PSMA-targeted PET imaging agent that enables visualization of localized prostate cancer as well as bone and soft tissue metastases to determine the presence or absence of recurrent and/or metastatic prostate cancer.

There is a significant unmet need for reliable, targeted imaging in prostate cancer, particularly in the high risk and biochemically recurrent populations, according to Lantheus.

Eton Eyes Epilepsy Drug Approval

  • Company: Eton
  • Type of Application: NDA
  • Candidate: Zonisamide Oral Suspension
  • Indication: epilepsy
  • Date: May 29

The NDA for zonisamide oral suspension for the treatment of partial seizures in patients with epilepsy was accepted for review in October. Eton recently announced that it has sold its neurology portfolio, including lamotrigine, zonisamide and topiramate to privately-held Azurity Pharma for up to $45 million, including upfront and milestone payments.

Azurity will assume control of all three products and will be responsible for commercialization following regulatory approval. Eton will support Azurity in the transition and through regulatory approval. Eton will also receive a single-digit percentage royalty on Azurity's net sales of the products.

Bristol-Myers Squibb's Zeposia On Track For Second Nod?

  • Company: Bristol-Myers Squibb
  • Type of Application: sNDA
  • Candidate: Zeposia
  • Indication: ulcerative colitis
  • Date: May 30

Zeposia is an oral drug, which was approved by the FDA for the treatment of adults with relapsing forms of multiple sclerosis in March 2020. Bristol-Myers Squibb is now looking to get FDA's approval to allow its usage as a treatment option for adults with moderately to severely active ulcerative colitis.

The company communicated FDA's acceptance of the sNDA on Feb. 1. Following the redemption of a Priority Review Voucher with the submission, the FDA has assigned a PDUFA goal date of May 30.

Will Roche's Esbriet Get The Nod For Another Lung Disease?

  • Company: Roche Holding AG RHHBY
  • Type of Application: sNDA
  • Candidate: Esbriet
  • Indication: unclassified interstitial lung disease
  • Date: May (expected)

Esbriet is an oral medicine approved for the treatment of a lung disease called idiopathic pulmonary fibrosis. Roche has filed the regulatory application for expanding the label to include the indication of unclassified interstitial lung disease.

ILD broadly describes a diverse group of more than 200 types of rare pulmonary diseases. These share similar features, including cough and shortness of breath.

The FDA accepted the sNDA for priority review on Jan. 21, with Roche stating that the decision is expected in May.

Adcom Reviews

FDA's Arthritis Advisory Committee is scheduled to meet on May 6 to discuss ChemoCentryx, Inc.'s CCXI NDA for avacopan oral capsules, for the treatment of anti-neutrophil cytoplasmic antibody-associated vasculitis. The meeting is scheduled for between 10 a.m. and 4:15 p.m.

FDA's Endocrinologic and Metabolic Drugs Advisory Committee will meet on May 27 to discuss Provention Bio, Inc.'s PRVB BLA for teplizumab intravenous infusion, for the delay or prevention of clinical type 1 diabetes mellitus in at-risk individuals. The regulatory agency 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.

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