Drug updates: January

Around 1500 people in England with spinal muscular atrophy (SMA), a rare genetic condition, will benefit from a new medicine after The National Institute for Health and Care Excellence (NICE) released draft guidance recommending risdiplam (also known as Evrysdi and manufactured by Roche) as part of a managed access agreement (MAA).

The MAA is a special agreement between NHS England, NHS Improvement, and the pharmaceutical company Roche. It will allow people to receive risdiplam treatment while more data is gathered to address uncertainties identified by the independent NICE committee. Following this, NICE will decide whether it should be recommended for routine use on the NHS and will update the guidance. Until then, it will be available through the MAA.

SMA is a progressive condition that affects the spinal cord nerves that control movement. This causes muscle weakness, progressive loss of movement, and breathing and swallowing difficulties.

SMA is classified into five major types (types 0 to 4). Type 1 SMA is the most severe form of SMA, and people with it usually die before the age of two if left untreated, usually because of respiratory failure.