FDA Blocks Ultomiris for NMOSD Treatment
AstraZeneca's efforts to expand the use of its long-acting complement C5 inhibitor, Ultomiris (ravulizumab), have hit a snag. The U.S. Food and Drug Administration (FDA) has issued a complete response letter (CRL) regarding the drug's application for treating neuromyelitis optica spectrum disorder (NMOSD), a rare autoimmune condition. The FDA's decision does not stem from a lack of clinical data but rather from concerns over the drug's risk evaluation and mitigation strategy (REMS).
FDA's Concerns and Required Modifications
The FDA has requested modifications to the REMS for Ultomiris, specifically focusing on ensuring patients' meningococcal vaccination status or the prophylactic administration of antibiotics before treatment. This request is based on data suggesting that meningococcal vaccines may offer incomplete protection against invasive meningococcal disease in patients receiving Ultomiris or its predecessor, Soliris. The Centers for Disease Control and Prevention (CDC) recommend considering antimicrobial prophylaxis for patients on these drugs to mitigate the risk of meningococcal disease.
AstraZeneca's Response and Future Steps
AstraZeneca has expressed its commitment to working closely with the FDA to address these concerns and bring Ultomiris to NMOSD patients in the United States as swiftly as possible. The company highlighted that Ultomiris has already received approval for NMOSD treatment in the European Union, Japan, and other countries. In the U.S., Ultomiris is approved for other indications, including paroxysmal nocturnal hemoglobinuria (PNH), hemolytic uremic syndrome (HUS), and generalized myasthenia gravis (gMG).
The Importance of Ultomiris for AstraZeneca
The development of Ultomiris as a successor to Soliris is of strategic importance for AstraZeneca, especially as Soliris is set to lose patent protection in the U.S. by 2025, opening the door to biosimilar competition. AstraZeneca has been actively transitioning patients from Soliris to Ultomiris, which offers the advantage of less frequent dosing—every eight weeks compared to every two weeks for Soliris. However, the competitive landscape is intensifying, with new treatments emerging for key indications like PNH, including Roche's crovalimab and Novartis' iptacopan.
Competitive Landscape in NMOSD Treatment
In the NMOSD space, Soliris faces competition from Horizon Therapeutics' Uplizna (inebilizumab) and Roche's Enspryng (satralizumab), both of which target different pathways involved in the disease. Despite the FDA's current decision, AstraZeneca remains optimistic about Ultomiris's potential to offer a valuable treatment option for NMOSD patients, pending the necessary adjustments to its REMS.
AstraZeneca's dedication to resolving the FDA's concerns underscores the company's commitment to advancing treatment options for rare diseases like NMOSD, while also navigating the challenges of a competitive and evolving pharmaceutical landscape.
