Attention Biotech Investors: Mark Your Calendar For April PDUFA Dates

A slew of drugs, including four new molecular entities, were approved in the month of March. NMEs are drugs that contain active moieties that have never been approved by the Food and Drug Administration previously and are indicators of innovation in drug research.

Among the notable approvals that came through in the month were Abecma, the first-ever cell therapy for a type of blood cancer; Fotivda, AVEO Pharmaceuticals, Inc.'s AVEO oral, next-generation therapy for treating advanced renal cell carcinoma; and KemPharm, Inc.'s KMPH Azstarys, a once-daily product for the treatment of attention deficit hyperactivity disorder in patients age six years and older.

PDUFA dates are binary events that invariably serve as make-or-break catalysts for stocks. These dates are deadlines by which the FDA reviews a new drug application before announcing its decision concerning the approvability/non-approvability of the drug.

Here are the key FDA approvals scheduled for April.

Acadia Hopes Against Hope to Get Label Expansion Psychosis Drug

• Company: ACADIA Pharmaceuticals Inc. ACAD
• Type of Application: supplemental new drug application
• Candidate: pimavanserin
• Indication: dementia
• Date: April 3

Pimavanserin, which is marketed under the trade name Nuplazid, has already been approved by the FDA for the treatment of hallucinations associated with Parkinson's disease psychosis. This time around, the company is seeking to get label expansion to include dementia-related psychosis.

An FDA communication the company disclosed March 8, nearly a month ahead of the scheduled PDUFA action date, poured cold waters on the company's hopes of a positive regulatory action.

FDA said it identified deficiencies that prevent discussion of labeling and post-marketing requirements. The agency, however, said the notification does not reflect a final decision on the information under review.

"Given the lack of feedback from the FDA to Acadia's multiple inquiries since the March 3 notification, a complete response letter is now the most likely outcome for pimavanserin by its April 3 PDUFA date," Raymond James analyst Danielle Brill said following the development.

Can Second Time Be Charm For Supernus?

• Company: Supernus Pharmaceuticals, Inc. SUPN
• Type of Application: NDA
• Candidate: SPN-812
• Indication: attention deficit hyperactivity disorder
• Date: early April

SPN-812, chemically viloxazine hydrochloride, is being evaluated for the treatment of ADHD in pediatric patients 6 to 17 years of age.

In early November, FDA issued a complete response letter for the original NDA, primarily citing issues related to the company's in-house laboratory that conducts analytical testing. The company said the lab had since then moved to a new location.

Subsequently, the company said in late February it received notice from the FDA that its resubmission for SPN-812 is considered a Class I resubmission.

Can Avenue's Pain Drug Soothe In Its Second Attempt At FDA's Clearance?

• Company: Avenue Therapeutics, Inc. ATXI, a Fortress Biotech, Inc. FBIO company
• Type of Application: NDA
• Candidate: intravenous tramadol
• Indication: moderate-to-severe pain
• Date: April 12

Intravenous tramadol is a potential alternative that could reduce the use of conventional opioids for treating acute pain. The FDA issued a CRL for the original application on Oct. 10, with the agency stating that IV tramadol, intended to treat patients in acute pain who require an opioid, is not safe for the intended patient population.

If a patient requires an analgesic between the first dose of IV tramadol and the onset of analgesia, a rescue analgesic would be needed, and the likely choice would be another opioid, the FDA had said. This, according to the agency, would result in opioid stacking and increase the likelihood of opioid-related adverse effects.

Following a Type A meeting with the FDA, the company resubmitted the NDA Feb. 12 and the agency subsequently deemed it as a complete response.

Sol-Gel Seeks Approval For Inflammatory Skin Disorder Treatment

• Company: Sol-Gel Technologies Ltd. SLGL
• Type of Application: NDA
• Candidate: Epsolay
• Indication: April 26
• Date: inflammatory lesions of rosacea

Epsolay, chemically benzoyl peroxide, is a topical cream that is being evaluated as a treatment option for inflammatory lesions of rosacea. Papulopustular rosacea is a chronic and recurrent inflammatory skin disorder that affects nearly 5 million Americans.

Can Protalix Finally Get The Nod After 3-Month Delay?

• Company: Protalix BioTherapeutics, Inc. PLX
• Type of Application: Biologic License Application
• Candidate: pegunigalsidase alfa
• Indication: Fabry Disease
• Date: April 27

The BLA submitted by Protalix, along with its development and commercialization partner Chiesi, was accepted for priority review in August 2020 and the PDUFA date was set for Jan. 27. With COVID-19 constraining FDA's ability to carry out facility inspections, the review period was extended by three months.

Fabry disease is a rare genetic disorder that prevents the body from making an enzyme called alpha-galactosidase A, which helps our body to break down a type of fat for use by our body. Currently, it's being treated with enzyme replacement therapy, which would mean replacement of the enzyme through intravenous infusion once every week.

Pegunigalsidase alfa is designed to be a plant cell culture-expressed, and a chemically modified version of, the recombinant alpha-Galactosidase-A protein.

Go or No-go For Ardelyx's Drug to Treat Elevated Blood Phosphate Levels?

• Company: Ardelyx, Inc. ARDX
• Type of Application: NDA
• Candidate: tenapanor
• Indication: Hyperphosphatemia
• Date: April 29

Tenapanor is being evaluated for the control of phosphorus in the blood in adult patients with chronic kidney disease on dialysis. It's estimated to affect more than 745,000 dialysis patients in major developed countries.

Hyperphosphatemia is currently treated with phosphate binders, but without much success. Tenapanor is a non-binder therapy that targets the primary pathway of phosphorus absorption.

Can United Therapeutics Get Label Expansion For Drug to Treat High Blood Pressure In Lungs

• Company: United Therapeutics Corporation UTHR
• Type of Application: sNDA
• Candidate: Tyvaso
• Indication: pulmonary hypertension associated with interstitial lung disease
• Date: April (expected)

Tyvaso is treprostinil inhalation solution that is awaiting regulatory clearance for the treatment of pulmonary hypertension associated with interstitial lung disease. If approved, it would be the first and only therapeutic approved for the treatment of PH-ILD, a condition that afflicts approximately 30,000 people, the company said.

Tyvaso is currently indicated for the treatment of pulmonary arterial hypertension to improve exercise ability.

Pfizer Awaits Nod For Skin Inflammation Treatment

• Company: Pfizer Inc. PFE
• Type of Application: NDA
• Candidate: abrocitinib
• Indication: moderate to severe atopic dermatitis
• Date: April

Abrocitinib, being evaluated in both 100mg and 200mg dose formulations, is an investigational oral once-daily Janus kinase 1 inhibitor, for the treatment of moderate to severe atopic dermatitis in patients 12 and older. Atopic dermatitis is a chronic skin disease characterized by inflammation of the skin. It's one of the most common, chronic, relapsing childhood dermatoses, affecting up to 10% of adults and up to 20% of children worldwide

"Abrocitinib has demonstrated strong efficacy at relieving the signs and symptoms of atopic dermatitis, including rapid reduction of itch, across multiple clinical trials. If abrocitinib is approved, it could make a meaningful difference in real-world clinical practice," said Jonathan Silverberg, a clinical dermatologist at the George Washington University School of Medicine and Health Sciences.

Adcom Calendar

FDA's Circulatory System Devices Panel of the Medical Devices Advisory Committee will meet April 6 to discuss TransMedics Group, Inc.'s TMDX premarket approval application for the TransMedics Organ Care System Heart.

The heart system is a portable extracorporeal heart perfusion and monitoring system indicated for the resuscitation, preservation, and assessment of donor hearts in a near-physiologic, normothermic, and beating state intended for a potential transplant recipient.