SMA Europe has launched an alliance that is demanding that, by 2025, newborn screening programs in Europe include a test for spinal muscular atrophy (SMA).

The new European Alliance for Newborn Screening in Spinal Muscular Atrophy (SMA NBS Alliance) was launched as a final initiative to mark this year’s SMA Awareness Month.

Its members include the 19 patient organizations that are part of SMA Europe, as well as EURORDIS — Rare Diseases Europe, the European Alliance of Neuromuscular Disorders Associations, TREAT-NMD, and pharmaceutical companies AveXis — now Novartis Gene Therapies — Biogen, and Roche, SMA Europe said in a press release.

To help achieve its goal of having every newborn in Europe tested for the neurodegenerative disorder, the members of the SMA NBS Alliance will take part in a variety of activities explaining why SMA should be included in national screening programs. These activities include writing a white paper, or an in-depth report on the subject.

In addition, members are organizing activities in their respective countries aimed at getting health regulators to consider adding SMA to newborn screening panels. National screening committees regularly review screening recommendations for various conditions as new evidence becomes available.

About newborn screening

Newborn screening (NBS) is the practice of testing all babies in their first days of life for certain disorders that are treatable, but difficult or impossible to detect clinically. This allows for the identification of patients before the first symptoms emerge.

National screening committees regularly review their recommendations on screening for different conditions in light of new research and evidence becoming available. We hope that with newly launched treatments, reviews will now take place and following new applications, revised guidance will be delivered across countries to include SMA.

A chance for a normal life

Newborn screening allows to treat children with SMA, before motor neuron destruction, thus giving them a chance of a normal life.

We hope that with newly launched treatments, reviews will now take place and following new applications, revised guidance will be delivered across countries to include SMA. Newborn screening allows to treat children with SMA, before motor neuron destruction, thus giving them a chance of a normal life", stated SMA Europe.

Two SMA treatments are approved in Europe

Now, two SMA treatments are approved in Europe that increase the body’s production of SMN, a protein essential to motor neuron survival. Those treatments are Spinraza (nusinersen) by Biogen, and Zolgensma, a gene therapy developed by AveXis, a Novartis company.

A third treatment, Evrysdi (risdiplam), developed by Roche and Genentech, has been approved in the U.S., and a filing is expected to be made shortly to the European Medicines Agency (EMA), the regulatory agency for the European Union.