Alexion’s Soliris follow-up Ultomiris snares EU approval

Alexion has the EU approval it needed for Ultomiris, a follow-up to its rare disease therapy Soliris that will lose patent protection in Europe next year.

The European Commission has approved Ultomiris (ravulizumab) for paroxysmal nocturnal haemoglobinuria (PNH), a rare and potentially fatal blood disorder that is one of Soliris’ main indications. The US FDA gave the green light to the new drug in PNH last December.

Ultomiris has the same complement C5 target as Soliris (eculizumab), interrupting a pathway leading to the destruction of red blood cells, but is longer-acting as it is dosed as an infusion every eight weeks versus every two weeks for its predecessor.

Analysts at Clarivate think that profile could drive sales to almost $2 billion in 2023, helping to plug the gap when Soliris’ $3.5 billion sales start to face biosimilar competition from next year in Europe and in 2021 in the US, although Alexion thinks it may be able to protect its US franchise until 2027.

Ultomiris has been cleared by the EMA to treat patients with haemolysis with clinical symptoms indicative of high disease activity, as well as for those who are clinically stable after having been treated with Soliris for at least the past six months.

PNH is an ultra-rare and severe disease that, when left untreated, may cause a wide range of debilitating symptoms and complications, including blood clots that can lead to organ damage, stroke, heart attack, and death.

Soliris is also approved to treat atypical haemolytic uremic syndrome (aHUS), generalised myasthenia gravis (gMG) and neuromyelitis optica spectrum disorder (NMOSD), and Alexion is also looking to extend the uses of Ultomiris beyond PNH as it tries to follow the success of its cash-cow product.

A phase 3 trial in NMOSD is just getting started, which if positive could add another $1 billion to Ultomiris’ sales potential by 2028, according to analysts at SVB Leerink.

Meanwhile Alexion has already filed approval of the new drug in aHUS, with an FDA decision due in October, and has also started pivotal testing of the drug in gMG.

John Orloff, head of R&D at Alexion, said: “We believe Ultomiris will become the new standard of care for patients with PNH by providing immediate and complete C5 inhibition, sustained throughout the eight-week dosing interval, and reducing the number of infusions per year from 26 with Soliris to only 6 or 7 with Ultomiris.”

“We are also particularly pleased by the positive data showing patients can successfully transition from Soliris to Ultomiris,” he added.