Biotech Investors: August's Key PDUFA Catalysts You Must Know

Zinger Key Points
  • Prescription Drug User Fee Act, aka, PDUFA dates are key binary catalysts
  • FDA is mandated to announce its decision on a drug by its PDUFA date
  • The unfolding month’s calendar is relatively light
Biotech Investors: August's Key PDUFA Catalysts You Must Know

Regulatory decisions have yet to pick up pace this year. July’s calendar was light, but the good news was that most decisions turned out to be positive. That said, none of the drugs approved during the month were new molecular entities, underlining a lack of innovation going on in the sector.

The following treatments received Food and Drug Administration’s blessings in July:

Horizon Therapeutics Ltd.’s HZNP gout treatment Krystexxa got an expanded label to be used in combination with methotrexate

Incyte Corporation’s INCY Opzelura also received a label expansion for use in vitiligo, an autoimmune disorder that leads to the skin losing its pigmentation.

GlaxoSmithKline plc’s GSK kidney disease drug Benlysta was approved to treat lupus nephritis – a kind of kidney inflammation, in children, five to 17 years old.

The Rejections: The FDA poured cold water on BeiGene Ltd.’s BGNE plan to get its first non-China approval for its anti-PD-1 antibody Tisleizumab. The regulator deferred the decision on the drug, citing travel restrictions necessitated by COVID-19 that delayed inspections.

Related Link: As COVID Subsidies, Here Are 8 Infectious Diseases That Could Emerge As Potent Threats

Key regulatory decisions scheduled for August:

Acadia Looks Ahead To Nuplazid Label Expansion After Adcom Snub

Company: ACADIA Pharmaceuticals, Inc. ACAD
Type of Application: Nuplazid (pimavanserin)
Candidate: supplemental new drug application
Indication: Alzheimer’s disease psychosis
Date: August 4

The FDA’s Psychopharmacologic Drugs Advisory Committee which met in mid-June voted 9-3 that the evidence presented does not support the approval of pimavanserin for the treatment of hallucinations and delusions associated with Alzheimer’s disease psychosis. The stock pulled back about 33% following the adverse verdict.

Nuplazid was approved in the U.S. in 2016 for treating hallucination and delusions associated with Parkinson’s disease psychosis. It fetched $484.1 million in sales for this indication in 2021

The chances of the FDA clearing the drug for the additional indication is now slim. Incidentally, Acadia received a complete response letter for Nuplazid in dementia-related psychosis in April 2021.

Will Myovant’s 3-Month Wait Worth It?

Company: Myovant Sciences Ltd. MYOV
Type of Application: sNDA
Candidate: Myfembree
Indication: endometriosis-related pain
Date: August 6

Myovant co-develops Myfembree with pharma giant Pfizer, Inc. PFE, and it was originally approved by the FDA in May 2021 for managing heavy menstrual bleeding associated with uterine fibroids in premenopausal women, with a treatment duration of up to 24 months.

Myfembree fetched Myovant $4 million in sales in the June quarter.

The sNDA seeks to add another indication for Myfembree – this time around, to help manage moderate to severe pain associated with endometriosis. The original PDUFA date of May 6 was extended by three months by the drug regulator.

Related Link: Teladoc Health's Revised Outlook 'Implies More Downside' According To This Analyst

Can bluebird bio Snag First Of Two Approvals For The Year?

Company: bluebird bio, Inc. BLUE
Type of Application: BLA
Candidate: betibeglogene autotemcel (beti-cel)
Indication: β-thalassemia
Date: August 19

Hopes of a positive verdict gained ground after the FDA’s Cellular, Tissue, and Gene Therapies Advisory Committee voted unanimously, recommending approval of beti-cel as a one-time gene therapy to treat Beta-thalassemia patients dependent on blood transfusions.

After a 14% jump on June 13 in reaction to the verdict, the stock has come off and is now at its pre-Adcom level.

Contingent on the FDA approval coming through, the company plans a commercial launch in the fourth quarter. It estimates that about 1,500 addressable patients are available in the U.S., with the average lifetime medical care cost per patient at $6.4 million.

‘Go’ Or ‘No-Go’ For Amicus’ Pompe Treatment?

Company: Amicus Therapeutics, Inc. FOLD
Type of Application: NDA
Candidate: miglustat for AT-GAA
Indication: Pompe disease
Date: August 29

The original PDUFA date of May 29 was extended by three months to provide the FDA with additional time to review the information submitted by the company as part of its ongoing reviews.

AT-GAA is an investigational two-component therapy that consists of cipaglucosidase alfa, administered in conjunction with miglustat. It is being evaluated for an inherited lysosomal disorder called Pome disease.

The disease can be debilitating and is characterized by severe muscle weakness that worsens over time. It ranges from a rapidly fatal infantile form with significant impacts on heart function to a more slowly progressive, late-onset form primarily affecting skeletal muscle. It is estimated that Pompe disease affects approximately 5,000 to 10,000 people worldwide.

Can Second Time Be Charm For CorMedix?

Company: CorMedix, Inc. CRMD
Type of Application: NDA
Candidate: defencath
Indication: catheter-related blood stream infections
Date: August 29 (estimated)

Defencath is to be used as a catheter lock solution in hemodialysis patients for the prevention of catheter-related bloodstream infections. The original application was issued a CRL in March 2021.

CorMedix filed a resubmission in late February, and it was accepted as a complete Class 2 response, with a six-month review cycle, in late March. The new PDUFA date could potentially be on August 29.

J&J Awaits Nod For Blood Cancer Drug

Company: Johnson & Johnson JNJ
Type of Application: BLA
Candidate: teclistamab
Indication: multiple myeloma
Date: August (estimated)

Teclistamab is an investigational, off-the-shelf, T-cell redirecting, a bispecific antibody targeting both B-cell maturation antigen (BCMA) and CD3. An EMA Committee has recommended conditional approval for the treatment in Europe, where it is given the brand name Tecvayli.
If approved, teclistamab could be the frontrunner in the BCMA T-cell engager space, Evaluate Pharma said. Pfizer, Regeneron Pharmaceuticals, Inc. REGN and Amgen, Inc. AMGN are also in the fray, it added.

Pending Decisions:

Axsome Therapeutics, Inc. AXSM: AXS-05 in major depressive disorder (Q2 schedule)

iShares Biotechnology ETF IBB ended Friday's session down 0.03% at $124.08, according to Benzinga Pro data.

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