Two new drugs for rare diseases from Biogen and BioMarin headed the list of new therapies backed by the EMA’s Committee for Medicinal Products for Human Use (CHMP).
Biogen closed in on EU approval of its spinal muscular atrophy (SMA) treatment Spinraza (nusinersen) in Europe, having already picked up a US approval for the highly-priced drug as 2016 drew to a close.
Spinraza is the first drug for the rare and often fatal genetic disease to be approved in Europe, which causes muscle weakness and progressive loss of movement. The only option for patients at the moment is supportive care, physiotherapy and mechanical ventilators. In the US Spinraza is priced at an eye-watering $750,000 in the first year of treatment, dropping to $375,000 thereafter, which Biogen says is justified by data showing it can improve survival and reduce the chances of patients that need to go on mechanical ventilation.
BioMarin got a green light for Brineura (cerliponase alfa), an enzyme replacement therapy for the ultra-rare neurodegenerative disease neuronal ceroid lipofuscinosis type 2 (CLN2), a form of Batten disease that is generally fatal by age 12. There is no approved drug to treat the underlying disease process and treatment efforts currently rely on managing symptoms.
BioMarin filed cerliponase alfa for approval in the US last July and after an FDA review extension, prompted by a request for more data, is now due to hear back from the agency later this week. The importance of Brineura to the company has gained prominence after it was forced to ditch Duchenne muscular dystrophy drug drisapersen in the face of regulatory resistance to approval.
There was also some much-needed good news for Sanofi and Regeneron, which claimed a positive opinion for their interleukin-6 (IL-6) inhibitor Kevzara (sarilumab) as a treatment for adults with moderate to severe rheumatoid arthritis. Kevzara was turned down by the US FDA last year because of manufacturing deficiencies but has since picked up an approval in Canada.
If approved by the EMA Kevzara will be the first direct rival in the EU to Roche’s big-selling RA therapy Actemra (tocilizumab), which brought in around $1.7bn last year with around a third of that total coming from Europe. Prior to the FDA rejection analysts were predicting big things for Sanofi and Regeneron’s drug, particularly after it outperformed AbbVie’s $14bn blockbuster Humira (adalimumab) in a head-to-head trial.
Sanofi has just picked up an FDA approval for eczema drug Dupixent (dupilumab) – which is made at the same facility as sarilumab – and the company is now hopeful of a US green light for the RA drug in the coming weeks.
In other news from the CHMP meeting, Pfizer picked up a positive opinion for Besponsa (inotuzumab ozogamicin) as a single-agent treatment for adults with relapsed or refractory CD22-positive B-cell precursor Philadelphia chromosome negative (Ph-) acute lymphoblastic leukemia (ALL) and Philadelphia chromosome positive (Ph+) ALL, who have previously failed treatment with at least one tyrosine kinase inhibitor (TKI).
The antibody drug conjugate (ADC), which targets CD22-positive cells and delivers the toxic compound calicheamicin into the interior of cells, is scheduled for a regulatory verdict from the US FDA in August. It was previously in development for non-Hodgkin’s lymphoma but failed a phase III trial.