![Hyung-jun Park, Professor of Neurology at Gangnam Severance Hospital, speaks at a press conference to commemorate Biogen Korea's "Spinraza" benefit coverage expansion on Feb. 2. [Photo: Reporter Eun Cho]](/news/photo/202311/144937_136755_4352.jpg)
Spinraza, a costly treatment for Spinal Muscular Atrophy (SMA) at 90 million won per dose, has had its benefit coverage criteria expanded. Patients with Type 3 SMA, who have developed symptoms after the age of 3, are now eligible for Spinraza.
Biogen Korea held a press conference on Nov. 2 and stated, “The benefit coverage criteria for Spinraza has been expanded, making it possible to treat SMA patients with onset after the age of 3.” The company stated, "We will continue to exert efforts towards enhancing the treatment environment for SMA.”
Previously, Spinraza was only covered for patients diagnosed with a deletion or mutation in the 5q SMN-1 gene, which makes the SMN protein, who were not on a permanent ventilator and had SMA-related clinical symptoms at age 3 or younger.
As of last month, the age requirement has been lifted, enabling Type 3b patients to receive Spinraza.
Spinal Muscular Atrophy is a rare genetic disorder characterized by the loss of motor neurons in the spinal cord and pituitary gland, leading to progressive muscle atrophy and weakness. It is caused by a deficiency in the production of the survival motor neuron (SMN) protein, and symptoms vary in severity.
The classification of Spinal Muscular Atrophy is based on age of onset and physical developmental milestones. Type 1 (0-6 months of age) involves an inability to sit or walk unassisted, with an average lifespan of two years. Type 2 (7-18 months of age) allows for independent sitting, but standing upright may not be possible, with approximately 70% surviving to age 25.
Spinraza is administered to more than 14,000 people with SMA in more than 60 countries around the world. It is an Antisense Oligonucleotide (ASO) with a mechanism of action that sustainedly increases the amount of SMN protein in the body and can be administered directly to the central nervous system, where motor neurons are located, through intrathecal injection therapy.
Dr. Hyung-jun Park, a neurology professor at Gangnam Severance Hospital, noted, "Patients with SMA type 3b suffer from progressive muscle weakness that hinders their daily lives, but because they are not covered by Spinraza, they have been relying on supportive care such as physical and rehabilitation therapy.” He continued, "With this benefit expansion, patients with Type 3b are expected to improve their motor function and quality of life by combining fundamental drug treatment."
Furthermore, he added, "Our Real World Evidence (RWE) studies conducted in multiple countries have demonstrated improvements in various motor function indicators in late-onset SMA patients receiving Spinraza."
[WIKI KOREA=Cho Eun, Reporter]
choeun@wikileaks-kr.org
