Innovator Eculizumab Wins 3 New US Patents, 1 New European Indication

Drug maker Alexion has announced 3 new US patents for its eculizumab (Soliris), a first-in-class monoclonal antibody that specifically binds to the complement protein C5. The new patents, directed to composition of matter, pharmaceutical formulations, and methods of treatment, will extend protection for the highly expensive drug until 2027. Eculizumab is approved by the FDA for the treatment of paroxysmal nocturnal hemoglobinuria (a rare, life-threatening disease of the blood characterized by destruction of red blood cells, presence of blood clots, and impaired bone marrow function) and atypical hemolytic uremic syndrome (a rare disease that causes abnormal blood clots to form in the small blood vessels in the kidneys).

“Alexion is the leader in complement biology and has invested decades of ground-breaking research and development in Soliris. We continue to strengthen our patent portfolio around this unique complement inhibitor,” said Ludwig Hantson, Alexion’s CEO. “These new patents work in concert with other patents and regulatory exclusivities to protect Soliris in all indications.” Soliris, with its list price of over $500,000 per patient per year, is among the most expensive drugs in the market. FiercePharma reports that the drug’s sales could reach $5.6 billion by 2020.

The 3 new patents protecting Alexion’s product may be designed to stave off competition from Amgen; the rival drug maker is currently developing a biosimilar eculizumab, ABP 959. Amgen recently completed data collection in a randomized, double-blind, single-dose, 3-arm, parallel group study conducted in Australia. The trial, conducted in 217 healthy male volunteers, seeks to demonstrate the pharmacokinetic similarity of biosimilar candidate ABP 959 and the reference eculizumab.

In addition to its new US patent protection, Alexion announced yesterday that it has gained the European Commission’s (EC) approval for a new indication for eculizumab. The EC granted an extension of the indication for eculizumab for the treatment of adult patients with refractory generalized myasthenia gravis (MG), a rare, autoimmune, neuromuscular junction disorder.

The EC approval follows review of data from the phase 3 REGAIN study, which did not meet its primary efficacy endpoint of change from baseline in the myasthenia gravis—activities of daily living profile (MG-ADL) total score.

Despite the fact that the study missed its endpoint, patient groups praised the results. Nancy Law, CEO of the Myasthenia Gravis Foundation of America, said, “People with MG need more and better treatment options, especially for those with refractory generalized MG, whose extreme muscle weakness can be devastating and even life-threatening. We look forward to advocating for and supporting patients with refractory MG who have a tremendous unmet need.”

Alexion has filed a supplemental Biologics License Application with the FDA, seeking an extension of Soliris’ indications for the treatment of MG.