The EC authorisation follows the positive recommendation by European Medicines Agency (EMA) Committee for Medicinal Products for Human Use (CHMP), which was based on the positive results from two clinical trials
Seat of the European Commission. (Credit: EmDee/Wikipedia)
Eiger BioPharmaceuticals has received the European Commission (EC) authorisation for Zokinvy (lonafarnib) to treat Hutchinson-Gilford progeria syndrome (HGPS) and processing-deficient progeroid laminopathies (PL).
Collectively referred to as ‘progeria’, HGPS and PL are severe ultra-rare and deadly paediatric diseases that cause extremely fast-paced ageing and premature death.
Zokinvy works to block the accumulation of defective progerin and progerin-like proteins, which leads to cellular instability and premature ageing in people with progeria.
The drug is indicated for patients aged 12 months and above, with a genetically confirmed HGPS or PL, associated with either LMNA or ZMPSTE24 mutation.
The EC authorisation follows the positive recommendation by European Medicines Agency (EMA) Committee for Medicinal Products for Human Use (CHMP), granted in May this year.
Its centralised marketing authorisation is valid in all 27 member states of the European Union (EU), along with Iceland, Liechtenstein, and Norway.
Eiger president and CEO David Cory said: “The European Commission’s approval of Zokinvy, Eiger’s first approval in Europe, is the culmination of years of dedication and commitment by the Eiger team and the Progeria Research Foundation, our partners in this pioneering endeavour.
“Eiger’s mission is to bring innovative medicines to underserved patients around the world. We are proud of this approval which further demonstrates our ability to partner with investigators and regulators to achieve that mission.”
The CHMP recommendation was based on the positive results from two clinical trials.
In the clinical trials, Zokinvy showed a reduction in the risk of death in children by 72% and extended the average life of children and young adults with HGPS by 4.3 years.
The UK’s Medicine and Healthcare products Regulatory Agency (MHRA) is currently reviewing the drug, under the European Commission Decision Reliance Procedure.
Eiger is a commercial-stage biopharmaceutical company engaged in the development of advanced therapies for the treatment of hepatitis delta virus and other serious diseases.
The company has obtained exclusive global rights to Zokinvy from Merck, which will not receive milestone payments and waived royalty obligations over a limited volume of sales.