Spinal Muscular Atrophy Treatment Makes Progress Across the Globe

Evrysdi, a treatment for spinal muscular atrophy (SMA), has started to make progress around the world in terms of approval. Brazil’s regulatory agency, National Health Surveillance Agency, has approved  the medication. This goes hand in hand with the New Drug Application (NDA) filed in Japan.

About SMA

Spinal muscular atrophy (SMA) is rare genetic order that is characterized by muscle weakness and degeneration. It affects one in every 10,000 people but can vary in severity from person to person. A mutation in the SMN1 causes this condition, and it causes the loss of motor neurons in the spinal cord and brain stem. Due to this loss, muscles weaken and atrophy, specifically the muscles used for walking, breathing, swallowing, crawling, sitting up, and moving the head.

The symptoms of this condition depend on the severity and type of SMA.

  • Type I is the most severe, and is diagnosed after birth. The symptoms of this type include developmental delay, the inability to sit up or move the head, and trouble with breathing and swallowing.
  • Type II is diagnosed in children between six and twelve months old, and they experience trouble with standing and walking.
  • Those who have type III are diagnosed between early childhood and adolescence and cannot walk or climb stairs independently.
  • Type IV occurs in adults over 30 and is the least severe type. People with this form of SMA usually have mild muscle weakness, problems with breathing, twitches, or tremors.

Once these symptoms are noticed, a diagnosis is confirmed through neurological examination and genetic testing. While there is no cure for SMA, there are treatments available. Physical therapy, braces, and surgery can all help to improve movement. Noninvasive respiratory support and tracheostomies can address issues with breathing while gastrostomy can help with eating. Spinraza is the first FDA approved treatment for SMA, and Zolgensma has also recently been approved.

Evrysdi Across the Globe

As of now, two countries have approved Evrysdi for the treatment of SMA: the US and Brazil. Brazil has only recently granted approval, and they did so about seven months after Roche filed their application. There are currently fifteen countries who have already received an NDA for this medication, including Australia, Russia, South Korea, and India.

Japan has just joined this list, as they accepted Roche’s NDA in late October. Their acceptance came after evaluation of the FIREFISH and SUNFISH trials. The former evaluated evrysdi in those with Type 1 SMA, while the latter studied its effect on those with Type 2 or Type 3.

Medical professionals and patients are excited by these developments, as SMA patients are in need of another treatment option. With the pandemic, the need for treatments is even more dire. Hopefully more countries approve of this treatment and allow it to reach more patients.

Find the source article here.

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