UK: Girl with a Rare Genetic Condition is Treated with the World's Most Expensive Drug
A 19-month-old girl is expected to live a normal life after being treated with revolutionary gene therapy.
Teddi Shaw, born in Northumbria, a small county in North East England, was diagnosed with a disease called Metachromatic leukodystrophy (MLD). It is a rare genetic condition that causes damage to the nervous system and organs. The chances of survival amongst children are extremely low.
However, there was a slim chance of survival if Teddi was treated with what is known to be the world's most expensive drug. Atidarsagene autotemcel, also known by the brand name Libmeldy, is a gene therapy treatment developed by the Biotech company Orchard Therapeutics.
The National Health Service, the Government department of England, intervened and signed an agreement with Orchard therapeutics to get the drug at a discounted price. The list price of the drug is £2.8 million.
Teddi became the first patient to be cured by the National Health Service. She is reportedly doing great. According to her mother, she has no signs of MLD, and she walks and runs a lot. However, her family is still facing heartbreak because Teddi's sister Nala was also diagnosed with MLD last year. Unfortunately, her condition is more advanced to be benefitted from this treatment.
Libmeldy inserts functional copies of a faulty gene into the patient's stem cells thus treating the deadly genetic condition of MLD.
(Input from various media sources)