spinal muscular atrophy Archives - Medical Design Sourcing

One-time gene therapy Zolgensma from Novartis shows lasting benefits for SMA patients

[Zolgensma image from Novartis]

A gene therapy for spinal muscular atrophy (SMA) patients is making waves. Novartis (NYSE:NVS) revealed new long-term data highlighting the durability of Zolgensma (onasemnogene abeparvovec) up to 7.5 years after a single treatment. The data comes from two long-term follow-up studies, LT-001 and LT-002, which examined a range of patient populations and demonstrated a positive overall benefit-risk profile. LT-001 is an ongoing 15-year long-term follow-up study of patients who completed the phase 1 START study. More than 3,000 children have been treated with Zolgensma in clinical trials, managed access programs and commercial settings.

Novartis expects Zolgensma to eventually generate $1.5–2 billion, according to BioPharma Dive. NVS shares were up 1 percent to $83.01 today after announcing the new data.

FDA approved Zolgensma (onasemnogene abeparvovec-xioi) in 2019 for chi…

Roche’s Evrysdi wins FDA approval in newborns with spinal muscular atrophy

Roche (SIX:RO, ROG; OTCQX:RHHBY) has announced that the FDA has approved a label extension to allow the use of Evrysdi (risdiplam) in babies younger than two months old with spinal muscular atrophy (SMA).

SMA is a genetic disease associated with progressive muscle wasting and weakness that affects roughly 1 in 11,000 babies.

The drug first won FDA approval in 2020 for adults and children at least two months old.

The label extension was based on interim efficacy and safety data from the RAINBOWFISH study in newborns.

That data demonstrated the majority of pre-symptomatic babies who received Evrysdi achieved developmental milestones related to sitting and standing. In addition, half of the babies were walking after 12 months of treatment.

“The approval of Evrysdi for pre-symptomatic babies is particularly important, as early treatment of SMA before symptoms start to arise can help babies to achieve motor milestones,” said Dr. Richard Finkel, …

Biogen plays up spinal muscular atrophy pipeline

As Biogen Inc. (Nasdaq: BIIB) continues to struggle to capitalize on the Alzheimer’s drug Aduhelm (aducanumab), the company is highlighting new data from its Spinraza (nusinersen) and spinal muscular atrophy (SMA) research program.

First approved in 2016, Spinraza is a survival motor neuron-2 (SMN2)-directed antisense oligonucleotide indicated to treat spinal muscular atrophy (SMA).

At the Muscular Dystrophy Association (MDA) Clinical & Scientific Conference held March 13-16, 2022), Biogen will describe the Phase 3b ASCEND trial design. The company recently announced that it had enrolled the first patient in ASCEND and continues to enroll participants in that study.

Biogen is also sharing data from the Phase 4 RESPOND trial, which is testing the safety and efficacy of Spinraza in infants and toddlers with unmet clinical needs after receiving Zolgensma (onasemnogene abeparvovec).

The company also will provide an update from the Phase 2 NURTURE …