Following the FDA’s familiar playbook with a flurry of Friday afternoon approvals, the European Medicines Agency has endorsed several new drugs, including Sanofi’s Xenpozyme, an enzyme replacement therapy for acid sphingomyelinase deficiency.
In all, EMA's Committee for Medicinal Products for Human Use (CHMP) signed off on five new medicines and six label extensions on Friday.
As for Sanofi’s approval, acid sphingomyelinase deficiency (ASMD) is a lysosomal storage disease that leads to a buildup of fatty substances that can cause brain and organ damage. It was formerly called Niemann-Pick disease. Children with the type A form of ASMD typically die by age 3. Those with type B have a life expectancy of 17 years.
Xenpozyme, which was developed using recombinant DNA technology, is infused every two weeks. It was shown in two trials to improve lung function and to reduce swelling of the liver and spleen.
The approval is for non-central nervous system disorders associated with ASMD. The European Commision will review the CHMP recommendation and is expected to make a final decision in a few months.
The med won its first approval in Japan in March. The drug carries a breakthrough designation in the U.S., but the FDA has extended its review by three months. The target date for approval now is Oct. 3.
Also making the grade in Europe is Eli Lilly and Incyte’s Olumiant for alopecia areata. Despite the drug being from the troubled JAK inhibitor class, there is much enthusiasm for it as there are few reliable treatments for alopecia areata. Approval in the United States is expected soon.
Eiger's Zokinvy to treat infants with Hutchinson-Gilford Progeria syndrome and PTC Therapeutics’ gene therapy Upstaza to treat severe aromatic L-amino decarboxylase deficiency also scored CHMP nods. Both are the first medicines sanctioned in Europe for use against these conditions.
Hutchinson-Gilford Progeria is a disorder that causes premature aging. It can be confirmed through genetic testing. Most commonly, it’s initially diagnosed by limited growth or facial features such as a receding chin, pointed nose and a loss of hair or body fat. Most children with HGPS die in their early teens due to cardiovascular complications. It occurs in roughly 1 of every 4 million births.
Zokinvy, which won approval in the U.S. in 2020 and is administered in pill form twice daily, helps slow the progression of the disease.
Severe aromatic L-amino decarboxylase deficiency, meanwhile, strikes infants before age 1, preventing the development of motor function. Those with AADC often die of organ malfunction before age 10. It affects 1 in 118,000 in Europe. Patients treated with Upstaza showed more head control and were able to sit unassisted.
The authorizations for Zokinvy and Upstaza are for “exceptional circumstances,” allowing patients access to medicines that can’t be approved using a standard route due to the lack of comprehensive data.
The EMA's committee also issued a thumbs-up for HEMA Biologics’ Cavenfacta for the treatment of bleeding with congenital hemophilia. The drug scored U.S. approval in 2020.
And it sanctioned Calliditas’ hybrid treatment Kinpeygo for the autoimmune kidney disorder primary immunoglobulin A neuropathy.
Aside from Olumiant's label expansion, Novartis' Cosentyx, Merck's Keytruda and AbbVie's Rinvoq scored label expansions in Europe, among others.