NeurologyLive® Year in Review 2023: Top Stories in Neuromuscular Disorders

The NeurologyLive^® staff was hard at work in 2023, 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 2023.

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 Designates Myasthenia Gravis Agent Rozanolixizumab Application for Priority Review

UCB Pharma announced that the FDA has granted priority review for the biologic license application (BLA) for its agent rozanolixizumab), a potential treatment for adults with generalized myasthenia gravis (gMG) who are anti-acetylcholine receptor (AChR) or anti-muscle-specific tyrosine kinase (MuSK) antibody positive. If the FDA approves the review, it could deliver significant improvements for the safety and effectiveness of the treatment, diagnosis, or preventative care for this patient population.

2. FDA Approves Omaveloxolone As First Treatment for Friedreich Ataxia

The FDA has approved omaveloxolone (Skyclarys), an agent developed by Reata Pharmaceuticals for the treatment of Friedreich ataxia in adults and adolescents aged 16 years and older, making it the first and only therapy approved for the indication.

3. Treg Suppressing Agent COYA 302 Shows Slowing of ALS Disease Progression

New data from a proof-of-concept study assessing Coya Therapeutics’ investigational agent COYA 302 showed that over a 48-week treatment period, patients with amyotrophic lateral sclerosis (ALS) showed amelioration in the progression of their disease. The therapeutic, designed to enhance proinflammatory T lymphocytes (Treg), successfully increased Treg suppressive function and was safe throughout the study.

4. FDA Accepts New Drug Application for Duchenne Therapy Vamorolone

According to a recent announcement, the FDA has accepted Santhera Pharmaceuticals and ReveraGen BioPharma’s new drug application (NDA) for its investigational agent vamorolone, a first-in-class dissociative steroid, for the treatment of patients with Duchenne muscular dystrophy (DMD).

5. Recombinant Human Growth Hormone, Testosterone Improves Ambulation, Disease Burden in Facioscapulohumeral Muscular Dystrophy

Using daily recombinant human growth hormone (rHGH) combined with testosterone injections every 2 weeks, findings from a single-center study showed that patients with facioscapulohumeral muscular dystrophy (FSHD) experienced improvements in ambulation, strength, muscle mass, and disease burden. Investigators concluded that placebo-controlled trials are needed to investigate and confirm the benefit of this therapeutic approach.

6. Vutrisiran Shows Benefit Across Baseline Severities in Phase 3 HELIOS-A Study

In a new post-hoc analysis of the phase 3 HELIOS-A study (NCT03759379) assessing vutrisiran (Amvuttra; Alnylam), an FDA-approved RNA interference therapeutic, the therapy demonstrated benefit in key measures, compared with external placebo, across all baseline polyneuropathy severities among patients with hereditary transthyretin-mediated (hATTR) amyloidosis. These results suggest patients who initiate vutrisiran earlier in their disease course would retain a higher level of neurologic function after 18 months, also highlighting the importance of early diagnosis and treatment.

7. FDA Approves Cipaglucosidase Alfa and Miglustat as First Two-Component Therapy for Pompe Disease

The FDA has approved Amicus Therapeutics’ combination of cipaglucosidase alfa-atga (Pombiliti) and miglustat (Opfolda) as the first and only 2-component treatment for patients with late-onset Pompe disease (LOPD). This new therapy, which was already previously approved in the European Union and United Kingdom, is indicated for adults with the disease weighing at least 40 kg and who are not improving on their current enzyme replacement therapy (ERT).

8. FDA Clears Phase 2 Study of CAR T-Cell Therapy KYV-101 in Myasthenia Gravis

Kyverna Therapeutics has announced that the FDA has cleared its investigational new drug (IND) application for KYV-101, a fully human CD19 chimeric antigen receptor (CAR) T-cell therapy, to be assessed in a phase 2 study of patients with myasthenia gravis (MG), an autoimmune disorder.

9. FDA Approves Complement C5 Inhibitor Zilucoplan as Treatment for Myasthenia Gravis

According to an announcement, the FDA has approved UCB Pharma’s investigational agent zilucoplan, a complement C5 inhibitor, to treat patients with myasthenia gravis (MG). Approved under the market name Zilbrysq, the subcutaneously delivered medication is indicated for those with acetylcholine receptor antibody positive (AChR-Ab+) generalized MG, further expanding the toolbox of options for patients with the disease.

10. FDA Approves Acadia's Trofinetide for Rett Syndrome

The FDA has approved trofinetide (Daybue; Acadia Pharmaceuticals) for the treatment of Rett syndrome in adult and pediatric patients 2 years of age and older. This treatment, a novel synthetic analog of the amino‐terminal tripeptide of IGF-1 designed to potentially reduce neuroinflammation and supporting synaptic function, is the first and only approved therapy for Rett syndrome, signifying a major breakthrough.