NeurologyLive® Year in Review 2024: Top Stories in Neuromuscular Disorders

The NeurologyLive^® staff was hard at work in 2024, covering clinical news and data readouts from all over the United States and beyond, across a number of key neurology subspecialty areas. Between the major study publications and FDA decisions, and traveling to societal conference sessions to conduct expert interviews, the team spent all year bringing the latest news and updates to the website's front page.

Among our key focus areas is neuromuscular disorders, a field that has experienced perhaps its most rapid period of progress in the past 2 years, with large steps forward in genetic medicine, in particular. Although major news items—such as first-time approvals or new guidelines—often appear among the top pieces our team produces, sometimes smaller stories reach those heights for other reasons, such as clinical impact and interest, or concerns about other facets of care, for example. Whatever the reason for the attention these stories got, their place here helps provide an understanding of the themes in this field over the course of 2024.

Here, we'll highlight some of the most-read content on NeurologyLive^® this year. Click the buttons to read further into these stories.

1. FDA Approves Takeda’s Immune Globulin Infusion, Gammagard Liquid, for Chronic Inflammatory Demyelinating Polyneuropathy

Following the approval of Takeda's Hyqvia, the FDA has approved the company's immune globulin (IG) infusion 10% (human) (Gammagard Liquid) as an intravenous immunoglobulin (IVIG) therapy to improve neuromuscular disability and impairment in adults with chronic inflammatory demyelinating polyneuropathy (CIDP).

2. Ferrer’s ALS Agent FAB122 Fails to Meet Primary, Secondary End Points

According to an announcement from Ferrer on the phase 3 ADORE trial (NCT05178810), FAB122, an investigational oral formulation of edaravone, did not meet primary or key secondary end points in patients with amyotrophic lateral sclerosis (ALS). The company also announced the open-label extension ADOREXT study (NCT05866926) will be now concluded, based on the lack of efficacy of FAB122.

3. Amylyx Pharmaceuticals Discontinues AMX0035 After Failing Primary End Point in Phase 3 PHOENIX Trial

According to an announcement, Amylyx Pharmaceuticals will voluntarily discontinue AMX0035 (Relyvrio) and remove it from the market in the United States and Canada based on negative topline data from its phase 3 PHOENIX trial (NCT05021536) that showed AMX0035 did not meet its primary end point of change in ALS Functional Rating Scale-Revised (ALSFRS-R).^1,2 Although AMX0035 will no longer be available for new patients with amyotrophic lateral sclerosis (ALS), those currently on therapy in the US and Canada who wish to stay on treatment and consult with their clinician can be transitioned to a free drug program.

4. FDA Approves Dose Expansion of Catalyst's Amifampridine for Lambert-Eaton Myasthenic Syndrome

The FDA has approved a supplemental new drug application (sNDA) for a higher maximum daily dose of Catalyst Pharmaceuticals’ potassium channel blocker amifampridine (Firdapse) for the treatment of adults and pediatric patients with Lambert-Eaton myasthenic syndrome (LEMS), a rare autoimmune neuromuscular disorder characterized primarily by muscle weakness of the limbs. The decision expands the dosage options to include daily dosing of 80 mg to 100 mg for patients with the disease weighing more than 45 kg.

5. FDA Approves Efgartigimod as New Treatment for Chronic Inflammatory Demyelinating Polyneuropathy

The FDA has approved Argenx's coformulation therapy efgartigimod alfa and hyaluronidase-qvfc (Vyvgart Hytrulo) as a new treatment for adults with chronic inflammatory demyelinating polyneuropathy (CIDP). The therapy is the first and only neonatal Fc receptor (FcRn) blocker approved for the treatment of CIDP, and will be available as a once-weekly, 30- to 90-second subcutaneous injection.

6. FDA Grants Traditional Approval to Elevidys Gene Therapy for Ambulatory DMD, Accelerated Approval for Nonambulatory Patients

Months after the FDA accepted and filed Sarepta Therapeutics’ efficacy supplement to the biologics license application for SRP-9001 (Elevidys), otherwise known as delandistrogene moxeparvovec-rokl, the agency has granted it traditional approval for ambulatory patients with Duchenne muscular dystrophy (DMD). With traditional approval, SRP-9001's indication has been expanded to include patients aged 4 years and older with DMD who have a confirmed mutation in the DMD gene.

7. Pfizer’s Duchenne Gene Therapy Fails to Meet Primary End Point in Phase 3 CIFFREO Trial

According to a announcement, fordadistrogene movaparvovec (previously known as PF-06939926), Pfizer’s investigational mini-dystrophin gene therapy, did not meet its primary end point of improvement in motor function among ambulatory patients with Duchenne muscular dystrophy (DMD) in the phase 3 CIFFREO study (NCT04281485). The company noted that it will continue to closely monitor all participants enrolled in CIFFREO, and will evaluate appropriate next steps for this program.

8. FDA Approves Cranbury’s Generic Version of Deflazacort to Treat Duchenne Muscular Dystrophy

According to an announcement, the FDA has approved a new generic version of deflazacort (Emflaza; PTC Therapeutics), a treatment indicated for patients 5 years of age and older with Duchenne muscular dystrophy (DMD). The new generic, which allows for greater access and improved cost-effectiveness, is from Cranbury Pharmaceuticals, a wholly-owned subsidiary of Tris Pharma.

9. FDA Approves Expanded Indication for Cerliponase Alfa to Treat CLN2 Disease in Children Under 3 Years

According to an announcement from BioMarin Pharmaceutical, the FDA has approved an expanded indication for cerliponase alfa (Brineura), a therapy for children with neuronal ceroid lipofuscinosis type 2 (CLN2 disease, or Batten disease), to treat patients of all ages, including those younger than 3. In addition, the therapy will now be available to all patients, including those who are symptomatic or asymptomatic

10. FDA Approves Zevra Therapeutics’ Arimoclomol as First Treatment for Niemann-Pick Disease Type C

According to an announcement, the FDA has granted approval of Zevra Therapeutics’ arimoclomol (Miplyffa), an oral medication, for the treatment of neurological symptoms associated with Niemann-Pick disease type C (NPC) in adults and children 2 years of age and older. The treatment, which is combined with the enzyme inhibitor miglustat, becomes the first drug approved by the agency to treat patients with this condition.