Recently the FDA designated Apitegromb for fast tracking as a treatment for SMA. We wanted to provide some additional information about this treatment, read on for more information and trial details.
Apitegromab is a novel investigational drug designed to treat spinal muscular atrophy (SMA), a rare genetic neuromuscular disorder characterised by progressive muscle weakness and loss of motor function. SMA is caused by the loss of or mutations in the survival motor neuron 1 (SMN1) gene, which is essential for the proper functioning of motor neurons. The severity of SMA varies depending on the levels of SMN protein produced by the backup gene, SMN2.
Apitegromab, developed by Scholar Rock, is a monoclonal antibody that targets the latent form of myostatin, a protein that regulates muscle growth. By inhibiting the activation of myostatin, Apitegromab promotes muscle growth and strength. This drug is intended to be complementary to existing SMA therapies that primarily focus on increasing SMN protein levels.
The FDA’s Fast Track designation is a program that aims to expedite the development and review of drugs intended to treat serious conditions and fulfill unmet medical needs. This designation enables more frequent communication with the FDA, provides access to priority review, and allows for rolling submissions of the drug’s marketing application. It ensures the drug receives enhanced attention and support, accelerating patient availability.
Apitegromab has shown promising results in early-phase clinical trials, demonstrating improvements in motor function, muscle strength, and other clinical outcomes for people with SMA. The drug is undergoing further clinical testing in a Phase 2/3 trial called TOPAZ, which aims to evaluate its safety, efficacy, and tolerability in SMA patients.
Given the Fast Track designation, Apitegromab’s development and review process is expected to be expedited, potentially leading to its approval and availability for SMA patients within the next few years. However, the exact timeline depends on the outcomes of the ongoing clinical trials and regulatory reviews. Once approved, it is anticipated that Apitegromab will be made available to patients outside of clinical trials, expanding access to this promising therapy for those living with SMA.
