The FDA experienced a busy September 2024, with regulatory decisions impacting a range of therapeutic areas, including diabetes management, nephrology, neurology, and pulmonology. Approvals spanned novel drug formulations, expanded indications for existing therapies, and breakthrough designations for investigational agents.
Diabetes Technology Advances
Embecta Corporation received FDA 510(k) clearance for its disposable patch pump for insulin delivery, designed for adults with type 1 and type 2 diabetes. The tubeless pump features a 300-unit insulin reservoir, developed based on feedback from patients and healthcare providers. This reservoir size is expected to meet the needs of 64% of adults with type 2 diabetes for three-day wear, compared to only 38% with a 200-unit reservoir. Ascensia and Senseonics also announced FDA clearance of the Eversense 365 system, the world’s first 1-year continuous glucose monitor (CGM).
Nephrology: Sparsentan Approved for IgAN
Travere Therapeutics announced that the FDA granted full approval for sparsentan (Filspari) to slow kidney function decline in adults with primary IgA nephropathy (IgAN). The decision was supported by two-year data from the Phase 3 PROTECT study, which demonstrated sparsentan's superiority over irbesartan in reducing proteinuria and preserving kidney function.
Neurology: First Treatments for Niemann-Pick Disease Type C
September marked a turning point in the treatment of Niemann-Pick disease type C (NPC) with the approval of two therapies: arimoclomol (Miplyffa) from Zevra Therapeutics and levacetylleucine (Aqneursa) from IntraBio. Arimoclomol, approved on September 20, is indicated for patients aged 2 years and older in combination with miglustat. Levacetylleucine was approved as a stand-alone therapy for neurological symptoms associated with NPC in patients aged 15 kg and older. The FDA granted breakthrough therapy designation to Simcere Pharmaceuticals’ edaravone and dexborneol sublingual tablets (Sanbexin) for the treatment of acute ischemic stroke (AIS).
Pulmonology: Dupilumab Expands Reach
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 also received approval for adolescents with chronic rhinosinusitis with nasal polyps.
Other Notable Approvals and Regulatory Actions
- Guselkumab (Tremfya) received approval for ulcerative colitis.
- Benralizumab (Fasenra) was approved for eosinophilic granulomatosis with polyangiitis (EGPA).
- Lebrikizumab-lbkz (Ebglyss) was approved for moderate-to-severe atopic dermatitis in patients aged 12 and older.
- Ascendis Pharma submitted a supplemental Biologics License Application to the FDA for TransCon hGH (lonapegsomatropin) for the treatment of adults with growth hormone deficiency (GHD).
These regulatory actions reflect the FDA's ongoing efforts to address unmet medical needs across a diverse range of conditions, from common diseases like diabetes and COPD to rare disorders like Niemann-Pick disease.
