Sobi's high-priority rare disease drug runs into a wall at EMA
The EMA has rebuffed Sweden-based Sobi’s pitch for emapalumab, shooting down high hopes for a $568 million drug.
Sobi said it will request a re-examination by the regulators, which will kick off a new review process that should produce a decision by the end of the year. The antibody is designed to treat primary hemophagocytic lymphohistiocytosis in children under 18 years old by targeting interferon gamma (IFNγ).
The drug, already approved in the US as Gamifant, is one of the stars of Sobi’s pipeline after it restructured its operations to focus on hematology and immunology.
The company first licensed it from Swiss biotech Novimmune in 2018, paying $50 million and promising $400 million in milestones before deciding to acquire the whole thing — complete with intellectual property rights, the team working on it and a priority review voucher. Under the new deal, milestones add up to $518 million.
“During the last years our team has gained a lot of experience in this rather complex disease area,” CEO Guido Oelkers said in a statement, as over 100 patients have been treated in the US.
He didn’t elaborate on what might have gone wrong in the application. Franco Locatelli, the principal investigator in the EU, was quoted saying he was “significantly surprised” about the EMA recommendation against approval.
“I had the privilege to observe that this monoclonal antibody, targeting the main cytokine involved in the disease pathophysiology, was well tolerated and effective in a large proportion of the patients, representing a model of precision medicine,” he said. “While US children have since almost 2 years the possibility to be treated with this novel, safe, highly effective and targeted therapy, the EMA decision paves the way for migratory health flows towards non-European Centers that can grant this treatment.”
In the release Sobi also reiterated its estimated peak sales target of over $500 million — which “remains unchanged regardless of an approval in Europe.”
Sales of Gamifant in the first six months of 2020 were $26.7 million. In 2019, the first year of launch, total sales were $61.3 million.
By Sobi’s estimate, the most important markets based on number of patients for HLH — a rare but severe disease that can result in fever, enlargement of the liver and spleen, enlarged lymph nodes, rashes and hospitalizations — are China, followed by the US, Europe and Japan.
It’s also initiating clinical studies for pre-emptive treatment of patients with risk factors of HSCT acute graft failure.