The World’s Most Expensive Drug Eyes Approval Stateside
Can drug makers justify multimillion dollar price tags?
Ally Shaw was hit with the worst kind of news a parent could receive. Both of her young daughters were diagnosed with a rare genetic disorder.
For her oldest daughter Nala, nothing could be done. Her disease would progress and she would be gone too soon. Prognosis for her youngest though, could prove completely different.
The girls were diagnosed with Metachromatic leukodystrophy (MLD), a group of disorders characterized by toxic buildup of fatty materials (sulfatides) in cells of the nervous system. In these disorders, a genetic mutation causes a deficiency in an enzyme (arylsulfatase A) critical in maintaining healthy nerve cells.
Patients born with MLD face a poor prognosis. Most children with the infantile form die by age 5. While bone marrow transplants can delay progression, there isn’t a cure.
Until now.
Nineteen month old Teddi, the younger of the two Shaw girls, qualified for a potentially life saving treatment.
A deal between The National Health System in the United Kingdom and Orchard Therapeutics would provide Teddi groundbreaking treatment with Libmeldy in the hope of curing her MLD.
