Labor Day marks the unofficial finish of summer season, and kids who haven’t already began faculty can be returning to school rooms quickly. As a result of many kids undergo from meals allergic reactions, EpiPens have turn out to be a staple in colleges, standing able to rapidly administer an epinephrine injection to counteract an allergic response. This educational yr, kids may have an alternate.
The FDA in August permitted the primary nasal spray epinephrine, a product from ARS Pharma that can be marketed as neffy. The regulatory choice for the two mg spray covers the remedy of kind I allergic reactions, which incorporates reactions attributable to meals, medicines, and bug bites. It could be utilized by adults in addition to kids who weigh 30 kilograms (66 kilos) or extra. San Diego-based ARS mentioned an utility searching for to broaden the approval to kids weighing lower than 30 kg is on observe for an FDA submission by the top of this yr.
Injectable epinephrine for kind 1 allergic reactions has been round since 1987. ARS developed neffy as an aqueous formulation of epinephrine and an ingredient that improves its bioavailability — how a lot of a drug is out there to offer its impact when it’s administered intranasally. The brand new approval comes almost a yr after the FDA turned down ARS’s utility for neffy. ARS’s submission had relied on human issue research that examined how individuals work together with the drug/system. The FDA requested for extra medical knowledge.
Neffy’s August approval got here almost two months forward of the October goal date for a regulatory choice, which William Blair analyst Tim Lugo mentioned was anticipated after the FDA instructed ARS that it meant to maneuver rapidly on the assessment and the company was underneath “important strain from affected person advocacy teams” after final yr’s rejection of the applying.
“We view the early approval as a constructive forward of the return-to-school season when many sufferers are anticipated to resume prescriptions,” Lugo mentioned in a analysis observe despatched to buyers.
Lugo mentioned ARS sees market alternative in sufferers who will swap to neffy from a at present obtainable epinephrine product in addition to sufferers who’ve let their epinephrine prescriptions lapse, together with these unwilling to hold or use a needle product. Long run, the corporate see alternative in tapping into an estimated 13.5 million individuals who have been identified with a situation that might be handled with epinephrine however haven’t but acquired a prescription.
In an investor presentation, ARS mentioned it expects to launch neffy later within the present quarter. Final week, the corporate introduced European Fee approval of the nasal spray. ARS mentioned this product, which can be offered in Europe underneath the model identify EURneffy, will launch by the top of this yr. EURneffy can be marketed by one other firm with an already established business footprint in Europe.
With summer season wrapping up, right here’s a recap of different latest regulatory information:
—Emergent BioSolutions smallpox vaccine ACAM2000 expanded its FDA approval to incorporate mpox, which is from the identical virus household as smallpox. In the course of the mpox outbreak of 2022, the FDA permitted use of the vaccine underneath an expanded entry investigational new drug utility. The extra approval comes amid an ongoing mpox outbreak in Central Africa.
—Three up to date Covid-19 vaccines now have the FDA’s blessing to be used through the upcoming respiratory an infection season. The regulator permitted and licensed up to date Covid-19 vaccines from Moderna and companions BioNTech and Pfizer. The brand new photographs handle the KP.2 pressure of the omicron variant of SARS-CoV-2. That’s near the KP.3.1.1 subtype that’s at present most prevalent within the U.S., in line with the Facilities for Illness Management and Prevention. The FDA beforehand instructed vaccine makers that photographs addressing the KP.2 pressure had been most well-liked, if possible, for the 2024-2025 vaccination season. Late final week, the FDA additionally licensed an up to date model of a protein-based Covid-19 vaccine from Novavax.
—Johnson & Johnson most cancers drug lazertinib, model identify Lazcluze, is now FDA-approved as a first-line remedy for regionally superior or metastatic non-small cell lung most cancers (NSCLC). The drug is a once-daily tablet designed to focus on epidermal progress issue receptor (EGFR) exon 19 deletions or exon 21 L858R substitution mutations, which should be detected by a companion diagnostic. For this indication, the approval specifies Lazcluze should be given together with Rybrevant, a J&J drug initially permitted in 2021 as a remedy for NSCLC with exon 20 insertion mutations to EGFR. In March, the FDA permitted Rybrevant as a first-line remedy for such cancers.
—As anticipated, the FDA turned down Regeneron Prescribed drugs’ utility for linvoseltamab as a remedy for superior instances of a number of myeloma. Regeneron forecast the rejection earlier this month throughout its convention name to debate second quarter 2024 monetary outcomes, disclosing that that the FDA flagged unresolved findings with a third-party producer of linvoseltamab, a bispecific antibody that targets the BCMA protein on a number of myeloma cells. These findings concern one other firm’s drug candidate and Regeneron believes that matter has been resolved. Nevertheless, one other FDA inspection is required earlier than the company can approve the drug.
—Regeneron did welcome a regulatory approval in Europe that marks the primary for its bispecific antibody platform. The European Fee permitted odronextamab, model identify Ordspono, as a remedy for follicular lymphoma or diffuse massive B-cell lymphoma that has superior after two or extra earlier strains of remedy. The drug is designed to bind to CD20 on most cancers cells and CD3 on T cells. Ongoing medical trials are evaluating this drug as a monotherapy and in combos as an earlier line of most cancers remedy.
—Liquidia acquired tentative FDA approval for Yutrepia as a remedy 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 remedy of PAH. In accordance with Liquidia, the FDA mentioned Yutrepia met all benchmarks for high quality, security, and efficacy. Nevertheless, remaining approval can’t be granted till the Might 23, 2025, when United Therapeutics’ patent for its dry powder formulation of treprostinil expires. Liquidia is difficult the FDA’s choice.
—The FDA lifted a partial medical maintain on an antibody drug conjugate (ADC) MediLink Therapeutics is growing in partnership with BioNTech. The partial maintain on the drug, BNT326/YL202, was introduced in mid-June after MediLink reported treatment-related opposed occasions. BioNTech mentioned along with danger mitigation measures, the research will proceed with doses no larger than 3 mg per kilogram, the place the protection profile was manageable and inspiring medical exercise was noticed. BioNTech and MediLink started their partnership on the HER-3-targeting ADC final yr.
—Incyte already has a presence in graft-versus-host illness with Jakafi. FDA approval of axatilimab, model identify Niktimvo, offers the corporate one other GVHD drug with a distinct strategy. Whereas Jakafi is a small molecule JAK inhibitor, Niktimvo is an antibody designed to dam a protein referred to as colony stimulating factor-1. FDA approval of the brand new drug covers the remedy persistent GVHD in adults after not less than two prior strains of remedy. Niktimvo was initially developed by Syndax Prescribed drugs. In 2021, Incyte and Syndax started a partnership on the drug; the 2 firms will share in commercialization of the remedy within the U.S. whereas Incyte will commercialize the product within the rests of the world.
—The FDA permitted Galderma’s nemolizumab, model identify Nemluvio, as a remedy for prurigo nodularis, a uncommon neuroimmune illness that results in persistent itch and the formation of nodules on the pores and skin. The antibody drug is designed to inhibit IL-31, a signaling protein related to a number of inflammatory situations. The drug can be underneath FDA assessment for atopic dermatitis; a regulatory choice is predicted later this yr.
—AstraZeneca most cancers drug Imfinzi expanded its permitted lung most cancers makes use of to incorporate the remedy of adults with resectable early-stage non-small cell lung most cancers (NSCLC). The newest approval covers use of the drug together with neoadjuvant chemotherapy earlier than surgical procedure and as an adjuvant monotherapy after surgical procedure. The drug’s first lung most cancers approval was in 2018 as a remedy for superior instances NSCLC. In 2020, the FDA permitted the drug for treating extensive-stage small cell lung most cancers.
—Ascendis Pharma received FDA approval for Yorvipath for hypoparathyroidism, a uncommon illness through which sufferers have low ranges of parathyroid hormone. Approval of the drug, an engineered model of parathyroid hormone administered as a once-daily injection, comes as Takeda Pharmaceutical plans to discontinue manufacturing of Natpara, the one different engineered parathyroid hormone drug at present obtainable. Ascendis plans to launch Yorvipath within the first quarter of subsequent yr.
—The FDA rejected Lykos Therapeutics’ utility searching for approval for midomafetamine (MDMA), a psychedelic drug the corporate had developed as a remedy for post-traumatic stress dysfunction (PTSD). In accordance with San Jose-based Lykos, the FDA requested the corporate to conduct one other Part 3 medical trial to additional research the protection and efficacy of the drug, echoing considerations raised throughout an June advisory committee assembly. Lykos plans to request an FDA assembly to ask for a reconsideration of the regulatory choice and to debate a possible resubmission of a brand new drug utility.
—Novartis drug iptcaopan, model identify Fabhalta, expanded its label with accelerated FDA approval within the uncommon kidney illness immunoglobulin A nephropathy. On this indication, the complement inhibitor will compete with Travere Therapeutics’ Filspari and Calliditas Therapeutics’ Tarpeyo. Fabhalta was first permitted final December as a remedy for the uncommon blood dysfunction paroxysmal nocturnal hemoglobinuria.
—The FDA delayed its regulatory choice for Humacyte’s engineered blood vessels. The company instructed Humacyte it wants extra time to finish its assessment of ATEV, the corporate’s bioengineered human tissue meant to function an alternative choice to harvesting a vein from a trauma affected person. Durham, North Carolina-based Humacyte mentioned it doesn’t but have a brand new goal date for a regulatory choice.
—Adaptimmune Therapeutics’ Tecelra grew to become the first engineered cell remedy permitted by the FDA as a remedy for a strong tumor. The accelerated approval covers the remedy of superior instances of synovial sarcoma. Tecelra is made by engineering a affected person’s T cells with a T cell receptor that acknowledges a goal on the within of a most cancers cell.
—GSK most cancers drug Jemperli broadened its label to cowl nearly all of endometrial most cancers instances. The immunotherapy was initially permitted in 2021 as a remedy for superior or recurrent endometrial most cancers that carries a specific genetic signature referred to as a mismatch restore deficiency. The newest approval expands use of the drug to sufferers with mismatch restore proficient/microsatellite secure tumors. Such instances characterize between 70% and 75% of endometrial most cancers sufferers. Jemperli’s FDA approval within the expanded indication covers use of the remedy together with chemotherapy.
Photograph from Flickr consumer US Division of Schooling
