Gene Therapy, Libmeldy, Not Recommended by NICE

In December 2020, Libmeldy, a stem cell gene therapy for the rare genetic disorder metachromatic leukodystrophy (MLD), was approved by the European Medicines Agency (EMA). The novel drug also received a Regenerative Medicine Advanced Therapy (RMAT) designation by the FDA in the US, showing potential for an expedited approval in the USA.(1) According to the FDA, a drug is eligible for an RMAT if the drug is a cell therapy; “intended to treat, modify, reverse, or cure a serious or life-threatening disease or condition” and shows “preliminary clinical evidence indicates that the drug has the potential to address unmet medical needs.”(2) 

MLD is a fatal and rare gene disorder that is caused by mutations in the ARSA gene. Patients with MLD can have neurological damage and development regression. The life expectancy for babies born with MLD can be 4-5 years of age, while those with juvenile MLD can only live up to 10-20 years after symptom onset.(3) 

However, in its most recent draft guidance, the National Institute for Health and Care Excellence (NICE) has stated that it does not recommend Libmeldy citing that the drug has uncertain long-term evidence and is not sufficiently cost-effective. 

Orchard Therapeutics, the manufacturer, during its earnings call last August stated it expected an estimated price of $3 - $3.5 million for the drug. This is significantly more expensive than the most expensive drug, Zolgensma, in the market with a $2.1 million price tag. 

Orchard Therapeutics had also mentioned “flexible payment arrangements” including “value-based payment options,” although that did not seem to have changed NICE’s decision in the latest draft guidance. 

While NICE’s decision is not final, the draft guidance has offered some insights on key considerations when assessing high cost groundbreaking therapies, and explores different reimbursement models to consider.

As Libmeldy is also seeking FDA approval in the US, it is time for plan sponsors to explore strategies for managing these high-cost gene therapies that will continue to be present in the drug pipeline.