Roche has secured FDA approval for Evrysdi (risdiplam), marking a significant advancement in the treatment of spinal muscular atrophy (SMA) with a more affordable oral therapy option. The approval covers treatment for adults and children aged two months and older, positioning the drug to compete with existing high-cost treatments in the market.
Clinical Efficacy and Treatment Benefits
Clinical trials demonstrated Evrysdi's effectiveness across multiple patient populations, encompassing Types 1, 2, and 3 SMA with varying disease severity. The drug achieved notable milestones, particularly in infant patients who gained the ability to sit independently for at least five seconds - a significant achievement not typically observed in the natural progression of the disease. Additionally, the treatment showed improved survival rates without permanent ventilation at both 12 and 23-month benchmarks compared to natural disease progression.
Innovative Administration and Mechanism of Action
Evrysdi operates by increasing the production of survival motor neuron (SMN) protein, which plays a crucial role in maintaining healthy motor neurons and movement functionality. As a liquid medication, it offers the convenience of daily administration either orally or via feeding tube, allowing for at-home treatment - a significant advantage for patients and caregivers.
Market Position and Pricing Strategy
Roche has implemented a weight-based pricing model for Evrysdi, with annual costs ranging from under $100,000 for younger patients to up to $340,000 for others. This pricing structure positions Evrysdi as a more accessible alternative to existing treatments: Biogen's Spinraza, which costs $750,000 in the first year and approximately $375,000 annually thereafter, and Novartis's Zolgensma, a one-time gene therapy priced at $2.1 million.
Treatment Availability and Distribution
The drug will be accessible to U.S. patients within two weeks of approval through Accredo Health Group, an Express Scripts specialty pharmacy, offering direct-to-home delivery services. This distribution model enhances accessibility and convenience for patients requiring long-term treatment.
The development of Evrysdi represents a collaborative effort between Roche, the SMA Foundation, and PTC Therapeutics, demonstrating the power of partnership in advancing treatment options for rare diseases.
