September proved to be a mixed month as far as drug approvals are concerned, with Mallinckrodt PLC MNK 0.01% and Aquestive Therapeutics Inc AQST 4.22% facing rejections.
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.
Among the more fortunate ones were GlaxoSmithKline plc GSK 1.39%, which got the nod for an expanded indication - hypereosinophilic syndrome. Bristol-Myers Squibb Co's BMY 0.75% leukemia drug also received FDA's blessings, and after several hiccups, Eton Pharmaceuticals Inc's ETON 8.48% out-licensed preservative-free allergic conjunctivitis ophthalmic solution managed to win FDA approval.
In another notable approval of the month, Roche Holdings AG's RHHBY 0.05% Gavreto was vetted to treat adults with metastatic RET fusion-positive non-small cell lung cancer.
Total new molecular entity, or NME, approvals thus far this year stand at 40 compared to 30 during the same period in 2019. An NME is a drug containing an active moiety that has never been approved by the FDA previously, and is therefore considered a measure of drug innovation.
Here are the key PDUFA dates for the month of October.
Avenue Awaits Clearance For Pain Drug
- Company: Avenue Therapeutics Inc ATXI 1.66%, a Fortress Biotech FBIO 0.25% company
- Type of Application: NDA
- Candidate: intravenous tramadol
- Indication: moderate-to-severe pain
- Date: Oct. 10
Intravenous tramadol is a potential alternative that could reduce the use of conventional opioids for treating acute pain.
The FDA accepted Avenue Therapeutics' NDA on Feb. 13, 2020, and assigned a PDUFA goal date of Oct. 10.
"With no AdCom or additional data requests by the FDA, we do not foresee any potential delays, including from the ongoing COVID-19 pandemic, and therefore continue to project IV tramadol's U.S. approved by its PDUFA date," H.C. Wainwright analyst Ed Arce said in a mid-August note.
The analyst expects a broad label without additional stipulations from a Risk Evaluation and Mitigation Strategy program or a restrictive DEA classification.
Will Zosano's Lead Drug Gain The Nod For Migraine?
- Company: Zosano Pharma Corp ZSAN 56.79%
- Type of Application: NDA
- Candidate: Qtrypta
- Indication: Migraine
- Date: Oct. 20
Qtrypta is Zosano's proprietary formulation of zolmitriptan delivered utilizing its proprietary intracutaneous microneedle system in development for the acute treatment of migraine. The company's intracutaneous microneedle system technology consists of titanium microneedles coated with drug that are designed to enable rapid systemic administration of therapeutics to patients.
The NDA for the investigational asset was accepted for a standard review by the FDA on March 4.
"We believe Qtrypta, if approved, has the potential to offer patients rapid, sustained pain relief with no recurrence," said Steven Lo, CEO of Zosano in the release announcing the FDA acceptance of the regulatory application.
Spectrum's Chemotherapy-induced Neutropenia Drug
- Company: Spectrum Pharmaceuticals, Inc. SPPI 2.21%
- Type of Application: BLA
- Candidate: SPI-2012
- Indication: chemotherapy-induced neutropenia
- Date: Oct. 24
SPI-2012, or eflapegrastim, which is to be marketed under the trade name Rolontis, is a long-acting granulocyte-colony stimulating factor. It's a novel molecule for the treatment of neutropenia, with the potential to compete in a worldwide market that exceeds $6 billion, Spectrum said in an investor presentation.
Neutropenia is a condition where neutrophil count is very low in the blood and it can be caused due to diseases that damage the bone marrow or certain medications, including chemotherapy.
Can Regeneron Get The Nod For Antibody Cocktail Treatment For Ebola?
- Company: Regeneron Pharmaceuticals Inc REGN 1.49%
- Type of Application: BLA
- Candidate: REGN-EB3
- Indication: Ebola viral infection
- Date: Oct. 25
REGN-EB3, an investigational triple antibody cocktail treatment for Ebola virus infection, developed using the same rapid response platform currently being leveraged to develop novel COVID-19 antibody therapy.
The FDA accepted the BLA for priority review on April 16.
Can Second Time Be Charm For Kala?
- Company: Kala Pharmaceuticals Inc KALA 2.13%
- Type of Application: NDA
- Candidate: Eysuvis
- Indication: Dry eye disease
- Date: Oct. 30
Eysuvis, or loteprednol etabonate ophthalmic suspension, is being evaluated for the short-term treatment of the signs and symptoms of dry eye disease.
Kala's original Eysuvis NDA was handed down a complete response letter in August 2019, with the FDA seeking positive data from an additional clinical trial. Following positive results from the STRIDE 3 Phase 3 trial, the company resubmitted the NDA in April. The company communicated FDA's acceptance of the resubmitted application on May 26.
Merck's Wonder Cancer Drug On Track to Snag Another Approval
- Company: Merck & Co., Inc. MRK 1.48%
- Type of Application: BLA
- Candidate: Keytruda
- Indication: classical Hodgkin's lymphoma, or cHL
- Date: Oct. 30
The sBLA seeks approval for Merck's Keytruda as a monotherapy for the treatment of adult patients with relapsed or refractory cHL. The regulatory application was filed based on the results from the Phase 3 KEYNOTE-204 trial, in which Keytruda demonstrated a significant improvement in progression-free survival compared to Seattle Genetics, Inc.'s SGEN 1.12% brentuximab vedotin, a current standard of care in this patient population.
Adcom Calendar
A joint meeting of the Psychopharmacologic Drugs Advisory Committee and the Drug Safety and Risk Management Advisory Committee will review Alkermes' ALKS 1.99% NDA for olanzapine/samidorphan oral tablets for the proposed indications of schizophrenia and bipolar I disorder.
The committees will discuss the efficacy, safety, and benefit-risk profile of the combo treatment. The meeting is scheduled for Oct. 9.
