Highly Anticipated FDA Decision Comes Just in Time for Back-to-School Season

Labor Day marks the unofficial end of summer, and children who haven’t already started school will be returning to classrooms soon. Because many children suffer from food allergies, EpiPens have become a staple in schools, standing ready to quickly administer an epinephrine injection to counteract an allergic reaction. This academic year, children will have an alternative.

The FDA in August approved the first nasal spray epinephrine, a product from ARS Pharma that will be marketed as neffy. The regulatory decision for the 2 mg spray covers the treatment of type I allergic reactions, which includes reactions caused by food, medications, and insect bites. It may be used by adults as well as children who weigh 30 kilograms (66 pounds) or more. San Diego-based ARS said an application seeking to expand the approval to children weighing less than 30 kg is on track for an FDA submission by the end of this year.

Injectable epinephrine for type 1 allergic reactions has been around since 1987. ARS developed neffy as an aqueous formulation of epinephrine and an ingredient that improves its bioavailability — how much of a drug is available to provide its effect when it is administered intranasally. The new approval comes nearly a year after the FDA turned down ARS’s application for neffy. ARS’s submission had relied on human factor studies that tested how people interact with the drug/device. The FDA asked for more clinical data.

Neffy’s August approval came nearly two months ahead of the October target date for a regulatory decision, which William Blair analyst Tim Lugo said was expected after the FDA told ARS that it intended to move quickly on the review and the agency was under “significant pressure from patient advocacy groups” after last year’s rejection of the application.

“We view the early approval as a positive ahead of the return-to-school season when many patients are expected to renew prescriptions,” Lugo said in a research note sent to investors.

Lugo said ARS sees market opportunity in patients who will switch to neffy from a currently available epinephrine product as well as patients who have let their epinephrine prescriptions lapse, including those unwilling to carry or use a needle product. Longer term, the company see opportunity in tapping into an estimated 13.5 million people who have been diagnosed with a condition that could be treated with epinephrine but have not yet received a prescription.

In an investor presentation, ARS said it expects to launch neffy later in the current quarter. Last week, the company announced European Commission approval of the nasal spray. ARS said this product, which will be sold in Europe under the brand name EURneffy, will launch by the end of this year. EURneffy will be marketed by another company with an already established commercial footprint in Europe.

With summer wrapping up, here’s a recap of other recent regulatory news:

—Emergent BioSolutions smallpox vaccine ACAM2000 expanded its FDA approval to include mpox, which is from the same virus family as smallpox. During the mpox outbreak of 2022, the FDA permitted use of the vaccine under an expanded access investigational new drug application. The additional approval comes amid an ongoing mpox outbreak in Central Africa.

—Three updated Covid-19 vaccines now have the FDA’s blessing for use during the upcoming respiratory infection season. The regulator approved and authorized updated Covid-19 vaccines from Moderna and partners BioNTech and Pfizer. The new shots address the KP.2 strain of the omicron variant of SARS-CoV-2. That’s close to the KP.3.1.1 subtype that is currently most prevalent in the U.S., according to the Centers for Disease Control and Prevention. The FDA previously told vaccine makers that shots addressing the KP.2 strain were preferred, if feasible, for the 2024-2025 vaccination season. Late last week, the FDA also authorized an updated version of a protein-based Covid-19 vaccine from Novavax.

—Johnson & Johnson cancer drug lazertinib, brand name Lazcluze, is now FDA-approved as a first-line treatment for locally advanced or metastatic non-small cell lung cancer (NSCLC). The drug is a once-daily pill designed to target epidermal growth factor receptor (EGFR) exon 19 deletions or exon 21 L858R substitution mutations, which must be detected by a companion diagnostic. For this indication, the approval specifies Lazcluze must be given in combination with Rybrevant, a J&J drug initially approved in 2021 as a treatment for NSCLC with exon 20 insertion mutations to EGFR. In March, the FDA approved Rybrevant as a first-line treatment for such cancers.

—As expected, the FDA turned down Regeneron Pharmaceuticals’ application for linvoseltamab as a treatment for advanced cases of multiple myeloma. Regeneron forecast the rejection earlier this month during its conference call to discuss second quarter 2024 financial results, disclosing that that the FDA flagged unresolved findings with a third-party manufacturer of linvoseltamab, a bispecific antibody that targets the BCMA protein on multiple myeloma cells. Those findings concern another company’s drug candidate and Regeneron believes that matter has been resolved. However, another FDA inspection is required before the agency can approve the drug.

—Regeneron did welcome a regulatory approval in Europe that marks the first for its bispecific antibody platform. The European Commission approved odronextamab, brand name Ordspono, as a treatment for follicular lymphoma or diffuse large B-cell lymphoma that has advanced after two or more earlier lines of therapy. The drug is designed to bind to CD20 on cancer cells and CD3 on T cells. Ongoing clinical trials are evaluating this drug as a monotherapy and in combinations as an earlier line of cancer treatment.

—Liquidia received tentative FDA approval for Yutrepia as a treatment for pulmonary arterial hypertension (PAH). Yutrupeia is an inhalable powder formulation of treprostinil, a drug that United Therapeutics markets in injectable, oral, and inhaled formulations for the treatment of PAH. According to Liquidia, the FDA said Yutrepia met all benchmarks for quality, safety, and efficacy. However, final approval cannot be granted until the May 23, 2025, when United Therapeutics’ patent for its dry powder formulation of treprostinil expires. Liquidia is challenging the FDA’s decision.

—The FDA lifted a partial clinical hold on an antibody drug conjugate (ADC) MediLink Therapeutics is developing in partnership with BioNTech. The partial hold on the drug, BNT326/YL202, was announced in mid-June after MediLink reported treatment-related adverse events. BioNTech said in addition to risk mitigation measures, the study will proceed with doses no higher than 3 mg per kilogram, where the safety profile was manageable and encouraging clinical activity was observed. BioNTech and MediLink began their partnership on the HER-3-targeting ADC last year.

—Incyte already has a presence in graft-versus-host disease with Jakafi. FDA approval of axatilimab, brand name Niktimvo, gives the company another GVHD drug with a different approach. While Jakafi is a small molecule JAK inhibitor, Niktimvo is an antibody designed to block a protein called colony stimulating factor-1. FDA approval of the new drug covers the treatment of chronic GVHD after at least two prior lines of therapy of systemic therapy in adults and children weighing at least 40 kg. Niktimvo was initially developed by Syndax Pharmaceuticals. In 2021, Incyte and Syndax began a partnership on the drug; the two companies will share in commercialization of the therapy in the U.S. while Incyte will commercialize the product in the rest of the world. [Updated to clarify prescribing information.]

—The FDA approved Galderma’s nemolizumab, brand name Nemluvio, as a treatment for prurigo nodularis, a rare neuroimmune disease that leads to chronic itch and the formation of nodules on the skin. The antibody drug is designed to inhibit IL-31, a signaling protein associated with multiple inflammatory conditions. The drug is also under FDA review for atopic dermatitis; a regulatory decision is expected later this year.

—AstraZeneca cancer drug Imfinzi expanded its approved lung cancer uses to include the treatment of adults with resectable early-stage non-small cell lung cancer (NSCLC). The latest approval covers use of the drug in combination with neoadjuvant chemotherapy before surgery and as an adjuvant monotherapy after surgery. The drug’s first lung cancer approval was in 2018 as a treatment for advanced cases NSCLC. In 2020, the FDA approved the drug for treating extensive-stage small cell lung cancer.

—Ascendis Pharma won FDA approval for Yorvipath for hypoparathyroidism, a rare disease in which patients have low levels of parathyroid hormone. Approval of the drug, an engineered version of parathyroid hormone administered as a once-daily injection, comes as Takeda Pharmaceutical plans to discontinue production of Natpara, the only other engineered parathyroid hormone drug currently available. Ascendis plans to launch Yorvipath in the first quarter of next year.

—The FDA rejected Lykos Therapeutics’ application seeking approval for midomafetamine (MDMA), a psychedelic drug the company had developed as a treatment for post-traumatic stress disorder (PTSD). According to San Jose-based Lykos, the FDA asked the company to conduct another Phase 3 clinical trial to further study the safety and efficacy of the drug, echoing concerns raised during a June advisory committee meeting. Lykos plans to request an FDA meeting to ask for a reconsideration of the regulatory decision and to discuss a potential resubmission of a new drug application.

—Novartis drug iptacopan, brand name Fabhalta, expanded its label with accelerated FDA approval in the rare kidney disease immunoglobulin A nephropathy. In this indication, the complement inhibitor will compete with Travere Therapeutics’ Filspari and Calliditas Therapeutics’ Tarpeyo. Fabhalta was first approved last December as a treatment for the rare blood disorder paroxysmal nocturnal hemoglobinuria.

—The FDA delayed its regulatory decision for Humacyte’s engineered blood vessels. The agency told Humacyte it needs more time to complete its review of ATEV, the company’s bioengineered human tissue intended to serve as an alternative to harvesting a vein from a trauma patient. Durham, North Carolina-based Humacyte said it does not yet have a new target date for a regulatory decision.

—Adaptimmune Therapeutics’ Tecelra became the first engineered cell therapy approved by the FDA as a treatment for a solid tumor. The accelerated approval covers the treatment of advanced cases of synovial sarcoma. Tecelra is made by engineering a patient’s T cells with a T cell receptor that recognizes a target on the inside of a cancer cell.

—GSK cancer drug Jemperli broadened its label to cover the majority of endometrial cancer cases. The immunotherapy was initially approved in 2021 as a treatment for advanced or recurrent endometrial cancer that carries a particular genetic signature called a mismatch repair deficiency. The latest approval expands use of the drug to patients with mismatch repair proficient/microsatellite stable tumors. Such cases represent between 70% and 75% of endometrial cancer patients. Jemperli’s FDA approval in the expanded indication covers use of the therapy in combination with chemotherapy.

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