A pharmaceutical ingredient that’s been a key element in nasal decongestants for many years is having its efficacy known as into query, and the FDA is now weighing whether or not to vary the compound’s regulatory standing — probably resulting in many extensively used over-the-counter chilly and allergy merchandise being pulled from pharmacy cabinets. However earlier than the FDA takes any motion, it needs to listen to what the general public thinks.
The ingredient, oral phenylephrine, by itself and together with different lively components, is in decongestants offered below quite a few model names, resembling Sudafed and Mucinex. All merchandise containing oral phenylephrine proceed to be accessible to shoppers for now. However on Thursday, the FDA proposed an order that might take away oral phenylephrine from its non-prescription medication pointers, known as the OTC Monograph. The regulator emphasised that this proposed order is because of lack of efficacy, not due to security.
At one time, phenylephrine was uncommon in decongestants as a result of pseudoephedrine was the popular lively ingredient. However that compound might be made into methamphetamine. Efforts to curb meth manufacturing led to the Fight Methamphetamine Act of 2005, which moved pseudoephedrine-containing medication behind pharmacy counters. Drugmakers responded by making extra merchandise with oral phenylephrine, which isn’t lined by this regulation.
Final 12 months, an FDA advisory committee mentioned phenylephrine and unanimously voted that present scientific knowledge don’t assist the ingredient’s efficacy as a nasal decongestant. The committee raised no issues concerning the security of oral phenylephrine when used on the advisable dose. The FDA now says it has carried out a complete assessment of all accessible security and efficacy knowledge for oral phenylephrine as a nasal decongestant, together with historic knowledge used to assist the compound 30 years in the past. Talking throughout a convention name with journalists Thursday, Theresa Michele, director of the FDA’s workplace of nonprescription medication, stated new knowledge had develop into accessible for the reason that advisory committee assembly, which the company additionally reviewed.
“At this level, primarily based on the accessible knowledge, and considering the recommendation of the advisory committee, we’re proposing that oral phenylephrine isn’t efficient to alleviate nasal congestion on the advisable dose set forth within the monograph,” Michele stated.
Phenylephrine can be an ingredient in some nasal spray decongestants. The FDA’s proposal doesn’t prolong to such merchandise. The proposed FDA order might be discovered right here. Public feedback could also be submitted right here (use docket quantity FDA-2024-N-4734 ) by way of Could 7, 2025. After contemplating the feedback, the company could subject a last order eradicating oral phenylephrine from its pointers, which might imply nasal decongestants might not include the compound. Michele stated the FDA would then present producers with time to both reformulate their medicines or take away oral phenylephrine-containing merchandise from the market.
Don’t let the autumn sniffles get you down. Right here’s a glance again at different latest regulatory information, together with U.S. and European actions on a number of novel drugs (alas, no new decongestants):
Choices for Medicine and Units
—The FDA wants extra time to assessment Organon drug Vtama as a therapy for atopic dermatitis. The corporate stated the regulator has pushed out the December goal date for a call by three months to March 12, 2025. Vtama is from Roivant Sciences subsidiary Dermavant, which Organon just lately acquired for $175 million up entrance. Beneath Dermavant, the topical drug gained FDA approval in 2022 as a therapy for plaque psoriasis.
—Novartis most cancers drug Scemblix expanded its FDA-approved makes use of to incorporate the therapy of newly identified Philadelphia chromosome-positive continual myeloid leukemia (Ph+ PML). The pill, a part of a category of medication known as STAMP inhibitors, initially landed accelerated FDA approval in 2021 as a therapy for superior circumstances of Ph+ PML.
—The FDA accredited Iterum Therapeutics antibiotic Orlynvah. The regulatory choice covers the therapy of uncomplicated urinary tract infections in girls who’ve restricted or no oral antibacterial therapy choices. The drug, whose major ingredient is sulopenem, is taken as a pill twice each day for 5 days.
—Astellas Pharma landed FDA approval for zolbetuximab, model title Vyloy, as a therapy for gastric or gastroesophageal junction adenocarcinoma. It’s the primary FDA approval for a drug concentrating on the most cancers protein claudin 18.2 (CLDN18.2). The regulatory choice particularly covers sufferers whose most cancers is destructive for a unique most cancers protein known as HER2. Medicine are already accessible for HER2-positive cancers.
—In different Astellas information, the Japanese pharma firm withdrew its European Medicines Company software for geographic atrophy drug avacincaptad pegol. The corporate stated it made the choice after discussions with the company’s Committee for Medicinal Merchandise for Human Use. The drug, administered as a once-monthly eye injection, gained FDA approval in geographic atrophy final 12 months and is marketed within the U.S. below the model title Izervay. Failure to safe approval in Europe will make it tougher for Astellas to recoup the $5.9 billion it spent to accumulate Iveric Bio, the corporate that developed the drug.
—Pfizer respiratory syncytial virus vaccine Abrysvo expanded its FDA approval to incorporate adults age 18 by way of 59, broadening the marketplace for the product. The preliminary FDA approval of Abrysvo final 12 months lined sufferers age 60 and older. It’s additionally accredited to be used by pregnant people as a maternal vaccine; antibodies produced by the mom confer safety towards RSV supposed to final from the newborn’s delivery as much as six months of age.
—Gilead Sciences’ Trodelvy will not be accessible within the U.S. for treating urothelial carcinoma, an aggressive bladder most cancers. After discussions with the FDA, the corporate determined to voluntarily withdraw the product, which had gained accelerated approval on this indication in 2021. In Could, the corporate reported Trodelvy failed its confirmatory Part 3 take a look at in superior urothelial carcinoma. The withdrawal in urothelial carcinoma doesn’t have an effect on drug’s accredited makes use of in breast most cancers.
—After earlier regulatory setbacks, AbbVie lastly landed FDA approval for Vyalev, a Parkinson’s illness drug/system mixture product that gives 24 hour subcutaneous infusion of the drugs foscarbidopa and foslevodopa. Final 12 months, the regulator turned down the product’s software, asking for extra details about the pump within the system. The FDA didn’t increase any questions on security or efficacy, nor did it ask for an additional medical trial. This previous June, the company once more turned down AbbVie’s software for points on the product’s third-party producer.
—There’s a brand new Pfizer drug for each hemophilia A and B. The FDA accredited the antibody drug marstacimab for stopping or lowering frequency of bleeding episodes in sufferers 12 and older. Pfizer will market the drug below the model title Hympavzi. As a once-weekly injectable treatment, Hympavzi will present an alternative choice to IV-infused hemophilia therapies.
—The FDA wants extra time to assessment Intercept Prescription drugs’ software in search of full regulatory approval of Ocaliva, which had acquired accelerated approval in 2016 for the uncommon liver illness main biliary cholangitis. Intercept, now a subsidiary of Alfasigma, stated the company didn’t present a brand new date for a regulatory choice.
—Alzheimer’s illness drug Leqembi hit a regulatory impediment in Australia, the place the Therapeutic Items Administration determined towards registering the treatment. Eisai stated it can request a reconsideration as permitted below Australian regulation. It’s the second setback for the drug in latest months. Over the summer season, the drug acquired a destructive opinion from a European Medicines Company committee involved concerning the danger of extreme unwanted side effects and restricted profit. Leqembi has approvals within the U.S., Japan, China, and several other different markets.
—The FDA accredited a Thermo Fisher Scientific companion diagnostic to Voranigo, a Servier Prescription drugs’ drug accredited over the summer season for treating sufferers whose glioma is pushed by mutations to IDH1 or IDH2 enzymes. The diagnostic, known as Ion Torrent Oncomine Dx Goal Check, identifies sufferers whose most cancers carries the genetic signature making them eligible for therapy with the Servier drug.
—Superior circumstances of non-small cell lung most cancers can now be handled with a wearable system. The FDA accredited Novocure’s Optune Lua, a wearable know-how that kills most cancers cells with electrical indicators administered by way of electrodes positioned on the pores and skin. Novocure’s “tumor-treating discipline” know-how was first accredited in 2011 as a therapy for a recurrent sort of mind most cancers.
—The FDA rejected Zealand Pharma’s dasiglucagon, a drug developed to forestall and deal with hypoglycemia in kids who’ve the uncommon illness congenital hyperinsulinism. In keeping with the Copenhagen-based biotech, the regulator cited points at a third-party producer. The FDA didn’t increase any issues concerning the drug’s medical knowledge or security.
Advisory Committee Outcomes
—An FDA advisory committee voted 11 to three that the advantages of Lexicon Prescription drugs’ sotagliflozin don’t outweigh its dangers when used as an adjunct to insulin to deal with adults with sort 1 diabetes and continual kidney illness. However some committee members expressed assist to be used of the drug in subpopulations of sufferers. Sotagliflozin, given the model title Zynquista, is anticipated to obtain an FDA choice by Dec. 20. Sotagliflozin was accredited final 12 months for the therapy of coronary heart failure and is marketed as Inpefa on this indication.
—The European Drugs Company’s Committee for Medicinal Merchandise for Human Use but once more advisable towards renewing the advertising and marketing authorization for Translarna, a PTC Therapeutics drug for Duchenne muscular dystrophy. Translarna acquired conditional advertising and marketing authorization in 2014. After the drug failed its Part 3 research, the committee twice advisable towards renewing the authorization. The most recent opinion comes after the EMA directed the committee to rethink, considering “the totality of proof.” The committee stated its reassessment “concluded that the effectiveness of Translarna has not been confirmed.”
—Stealth BioTherapeutics moved a step nearer to potential approval of the primary drug for Barth syndrome. An FDA advisory committee voted 10 to six that Stealth’s drug, elamipretide, was efficient at treating the ultra-rare mitochondrial dysfunction. The committee vote follows a number of regulatory setbacks, together with a 2021 refuse-to-file letter during which the FDA identified that elamipretide failed its medical trial. Stealth maintains there was enchancment in a subgroup of sufferers and the brief period of therapy is the explanation for the trial failure. An FDA choice is anticipated by Jan. 29.
What’s New in Covid-19
—The European Fee granted advertising and marketing authorization to Novavax’s up to date Covid-19 vaccine, Nuvaxovid. Authorization for the protein-based vaccine covers its use in these age 12 and older. The FDA licensed the up to date model of Novavax’s Covid-19 vaccine in September.
—In different Novavax Covid-19 information, the FDA pressed pause on the corporate’s plans for late-stage testing of a mix flu/Covid-19 vaccine in addition to a standalone influenza vaccine. In keeping with Novavax, the medical maintain stems from a report of motor neuropathy in a single affected person outdoors the U.S. who acquired the mixture vaccine in a Part 2 research. The trial was accomplished in July 2023 and the intense antagonistic occasion was reported in September. Novavax stated earlier Covid-19 and influenza trials confirmed no indicators of motor neuropathy. The corporate’s standalone Covid-19 vaccine is unaffected by the medical maintain.
—In Covid-19 and flu testing information, the FDA granted advertising and marketing authorization to a Healgen Scientific over-the-counter, at-home take a look at for each pathogens. The OTC Healgen Speedy Verify Covid-19/Flu A&B Antigen Check makes use of a pattern from a nasal swab. The authorization covers use of the product by individuals age 14 and older inside 5 days of the beginning of signs. For kids as younger as 2, the pattern have to be collected by an grownup.
Photograph: Patrick T. Fallon/AFP, by way of Getty Photographs
