FDA News Month in Review: September 2024

Welcome to our monthly recap of news and updates from the US Food and Drug Administration (FDA)!

For the September 2024 edition, we spotlight, in chronological order, 13 FDA decisions and 7 announcements of other regulatory news, including drug safety warnings, New Drug Application (NDA) acceptances, and other movements made toward label expansion.

Want to hear from the experts? Look for our Related Content links underneath blurbs for insight into how this decision might influence practice moving forward.

FDA Decisions

FDA Clears Embecta's Tubeless Patch Pump

On September 03, 2024, Embecta Corporation announced the FDA had granted 510(k) clearance for their disposable patch pump for insulin delivery in adults with type 1 and type 2 diabetes. The tubeless pump features a 300-unit insulin reservoir, designed based on feedback from type 2 diabetes patients and healthcare providers. This reservoir size is expected to benefit 64% of adults with type 2 diabetes for three-day wear, according to Embecta. In contrast, a 200-unit reservoir would only meet the needs of 38% of this population.

Related Content: Diabetes Dialogue: Lingo Availability and FDA Clearance for Embecta's Tubeless Patch Pump (Video Below)

FDA Approves Travere Therapeutics’ Sparsentan to Slow Kidney Function Decline in Adult Primary IgAN

On September 5, 2024, Travere Therapeutics announced the FDA granted full approval for sparsentan (Filspari) to slow kidney function decline in adults with primary IgA nephropathy (IgAN). This decision was based on two-year data from the phase 3 PROTECT study, which showed sparsentan's superiority over irbesartan in reducing proteinuria and preserving kidney function. The approval expands access to this non-immunosuppressive treatment for patients at risk of disease progression.

Related Content: Jonathan Barratt, PhD, FRCP: Sparsentan Full FDA Approval, Future IgAN Developments (Video Below)

FDA Approves Guselkumab (Tremfya) for Ulcerative Colitis

On September 11, 2024, Johnson & Johnson announced the FDA has approved guselkumab (Tremfya) for the treatment of adults with moderately to severely active ulcerative colitis (UC). Based on results from the phase 2b/3 QUASAR study, guselkumab demonstrated the ability to induce clinical and endoscopic remission in patients with inadequate response to conventional therapies or biologics. This approval makes guselkumab the first fully human, dual-acting monoclonal antibody targeting IL-23 and CD64 for UC treatment.

FDA Gives Authorization for First Over-the-Counter Hearing Aid Software Device

On September 12, 2024, the FDA announced its authorization of the Hearing Aid Feature, the first over-the-counter hearing aid software device. Based on a customizable approach, the feature amplifies sound for individuals aged ≥18 years with mild to moderate hearing impairment. Notably, the device is compatible with Apple AirPods Pro, offering a clinical-grade hearing aid experience. This authorization follows the FDA's efforts to increase access to affordable hearing aids, in line with regulations introduced in 2022.

FDA Approves Dupilumab for Adolescents with Chronic Rhinosinusitis with Nasal Polyps

On September 13, 2024, the FDA approved dupilumab (Dupixent) as an add-on maintenance therapy for individuals aged 12-17 years with chronic rhinosinusitis with nasal polyposis (CRSwNP). This expands the drug’s prior approval from June 2019 for adults aged 18 and older with the same condition. Dupilumab is a fully human monoclonal antibody targeting the interleukin-4 (IL-4) and interleukin-13 (IL-13) pathways, offering a non-immunosuppressant treatment option for those suffering from CRSwNP.

FDA Approves Lebrikizumab Treatment of Eczema Among Patients Aged 12 and Older

On September 13, 2024, the FDA approved lebrikizumab-lbkz (EBGLYSS) for adults and children aged 12 and older with moderate-to-severe atopic dermatitis not adequately controlled by topical treatments. This targeted IL-13 inhibitor offers a new first-line biologic treatment option, significantly improving skin clearance and reducing itch after 16 weeks of therapy in clinical trials. Lebrikizumab is expected to be available in the U.S. soon, following approvals in Europe and Japan, and is administered via injection with a specific dosing regimen.

Related Content: Responding to FDA Approval of Lebrikizumab for Atopic Dermatitis, with Andrew Alexis, MD, MPH (Video Below)

FDA Clears Eversense 365, Marking First 1-Year CGM Option in Diabetes

On September 17, 2024, the FDA cleared the Eversense 365 system, the world’s first 1-year continuous glucose monitor (CGM), for use in people with type 1 and type 2 diabetes aged 18 and older. This fully implantable CGM system provides a 12-month sensor lifespan, offering greater convenience and fewer interruptions compared to short-term CGMs. Eversense 365 also includes features such as a daily replaceable adhesive, a removable transmitter, and vibration alerts for added patient flexibility and comfort.

FDA Approves Benralizumab for Eosinophilic Granulomatosis with Polyangiitis

On September 18, 2024, AstraZeneca announced the FDA's approval of benralizumab for the treatment of adult patients with eosinophilic granulomatosis with polyangiitis (EGPA). This approval is based on data from the phase 3 MANDARA trial, which compared benralizumab to mepolizumab, the only other FDA-approved therapy for EGPA. The trial demonstrated that benralizumab achieved remission in 59% of patients, offering a new treatment option for those suffering from this rare and difficult-to-treat disease.

FDA Issues CRL to Vanda Pharmaceuticals’ Tradipitant for Gastroparesis Symptoms

On September 19, 2024, the FDA declined to approve the New Drug Application (NDA) for tradipitant for treating gastroparesis symptoms, issuing a Complete Response Letter (CRL) to Vanda Pharmaceuticals. Vanda criticized the CRL as dismissive of their evidence and noted that the FDA suggested additional studies with inconsistent design and duration, while turning down multiple requests for an expert advisory committee review.

FDA Approves Arimoclomol, First Drug for Niemann-Pick Disease, Type C

On September 20, 2024, the FDA approved Zevra Therapeutics' arimoclomol (Miplyffa) for treating Niemann-Pick disease type C (NPC), making it the first drug authorized for this condition. Arimoclomol is indicated for patients aged 2 years and older and is prescribed in combination with miglustat to address neurological symptoms associated with NPC. The approval followed a 12-month, randomized clinical trial involving 50 patients, which demonstrated that arimoclomol slowed disease progression as measured by the rescored 4-domain NPC Clinical Severity Scale.

FDA Approves Levacetylleucine for Niemann-Pick Disease Type C

On September 25, 2024, the FDA approved IntraBio's levacetylleucine (Aqneursa) as the first stand-alone therapy for neurological symptoms associated with Niemann-Pick disease type C (NPC) in patients aged 15 kg and older. This approval comes shortly after the agency authorized approval for Zevra Therapeutics' arimoclomol, marking a significant advancement for NPC treatment options. The decision was based on a clinical trial demonstrating that levacetylleucine significantly improved neurological symptoms and functional abilities within 12 weeks.

FDA Approves Xanomeline and Trospium Chloride (Cobenfy) for Schizophrenia

On September 26, 2024, the FDA approved xanomeline and trospium chloride capsules (Cobenfy) for the treatment of schizophrenia, marking the first new pharmacological approach to this mental illness in over 40 years. Unlike traditional antipsychotics that target dopamine receptors, this dual M1/M4 muscarinic acetylcholine receptor agonist offers a new mechanism of action, addressing positive, negative, and cognitive symptoms of schizophrenia. Clinical trials demonstrated significant symptom reduction and improved long-term outcomes, with fewer adverse effects commonly associated with other antipsychotic medications.

FDA Approves Dupilumab (Dupixent) for Treatment of COPD

On September 27, 2024, the FDA approved dupilumab (Dupixent) for treating chronic obstructive pulmonary disease (COPD), providing a new biologic option for patients with uncontrolled symptoms. This decision, based on data from the BOREAS and NOTUS trials, marks dupilumab as the first biologic targeting interleukin-4 and interleukin-13 in COPD management, demonstrating significant reductions in exacerbations and improvements in lung function. Dupilumab now has over a dozen FDA approvals for various conditions, indicating its role in addressing type 2 inflammation across multiple diseases.

Other Regulatory News

Nanoscope Reports Positive End-of-Phase 2 Meeting for MCO-010 Gene Therapy

On September 12, 2024, Nanoscope Therapeutics received positive feedback from the FDA regarding MCO-010 gene therapy, which aims to treat severe vision loss due to Stargardt disease, allowing them to advance to a Phase 3 registration trial. The FDA confirmed that changes in best-corrected visual acuity (BCVA) will be the primary efficacy endpoint for this trial, which will be the first randomized, controlled gene therapy study for Stargardt disease. Encouraged by the promising results from the Phase 2 STARLIGHT trial, Nanoscope is optimistic about the potential of MCO-010 to restore vision in patients in Phase 3.

FDA Warns of Liver Injury Risk from Fezolinetant for Hot Flashes

On September 16, 2024, the FDA has issued a warning regarding the rare risk of serious liver injury associated with fezolinetant (Veozah), a treatment for moderate to severe vasomotor symptoms due to menopause. Following a postmarketing report of elevated liver blood test values and symptoms in a patient after 40 days of treatment, the FDA advises discontinuing the medication to prevent worsening liver damage. Updated recommendations now include more frequent liver blood testing for patients, with monthly evaluations for the first two months, and an immediate halt of the drug if any signs of liver injury occur.

FDA Grants Breakthrough Therapy Designation to Plozasiran for FCS

On September 16, 2024, the FDA granted Breakthrough Therapy designation to investigational plozasiran, as an adjunct to diet to lower triglycerides in adults with familial chylomicronemia syndrome (FCS).Plozasiran is a first-in-class RNA interference therapeutic designed to reduce apolipoprotein C-III (APOC3) production, which contributes to elevated triglyceride levels. Arrowhead Pharmaceuticals announced plans to submit a NDA by the end of 2024.

Obeticholic Acid’s Future in PBC After FDA Advisory Committee Meeting

A recent FDA Gastrointestinal Drug Advisory Committee meeting raised concerns about the benefit-risk profile of obeticholic acid (Ocaliva) for treating primary biliary cholangitis (PBC), leading to a negative opinion on its approval as a second-line treatment. The committee voted overwhelmingly against the drug's clinical benefits and expressed doubts about its risk profile, especially as new options like elafibranor and seladelpar have emerged. With a critical PDUFA action date approaching, there are fears that patients reliant on obeticholic acid may be left without effective treatment options if the FDA follows the committee's recommendation.

Related Content: Nancy Reau, MD: Obeticholic Acid’s Future in PBC After FDA Advisory Committee Meeting (Video Below)

FDA Accepts NDA for Delgocitinib Cream Treatment of Chronic Hand Eczema

On September 23, 2024, LEO Pharma announced the FDA accepted its NDA for delgocitinib cream, a potential treatment for chronic hand eczema (CHE), which could become the first option for adults in the U.S. with moderate to severe cases. Delgocitinib is a pan-Janus kinase inhibitor targeting the JAK-STAT signaling pathway, which plays a crucial role in the disease's pathogenesis. The NDA acceptance is based on successful Phase 3 trials (DELTA 1 and DELTA 2) showing significant improvements in the Investigator’s Global Assessment for patients using the cream compared to a vehicle.

FDA Accepts sNDA for Roflumilast Foam as Scalp, Body Psoriasis Treatment

On September 24, 2024, the FDA accepted the Supplemental NDA for roflumilast foam (ZORYVE®) to treat scalp and body psoriasis in adults and adolescents aged 12 and older. Supported by data from the pivotal ARRECTOR Phase 3 trial, roflumilast foam showed significant improvements in psoriasis symptoms compared to a vehicle, with more than 66% of participants achieving success in the Scalp-Investigator Global Assessment at Week 8. Roflumilast foam, a once-daily phosphodiesterase-4 (PDE4) inhibitor, was granted a target action date of May 22, 2025.

TransCon hGH Aims for FDA Label Expansion for Adult Growth Hormone Deficiency

On September 30, 2024, Ascendis Pharma submitted a supplemental Biologics License Application (sBLA) to the FDA for TransCon hGH (lonapegsomatropin) to treat adults with growth hormone deficiency (GHD). The submission was based on results from the Phase 3 foresiGHt trial, which showed that weekly TransCon hGH significantly improved primary and key secondary efficacy endpoints compared to placebo. The treatment addresses an unmet medical need, as many adults with GHD are currently untreated.