Novartis has reported positive topline outcomes from the multicentre Phase IIIB APPULSE-PNH trial of oral Fabhalta (iptacopan) for treating adults with paroxysmal nocturnal haemoglobinuria (PNH).

AstraZeneca announced Monday that it has secured FDA approval for an add-on to its blockbuster rare disease franchise, providing a potential differentiator as it tries to hold off competition from Novartis, Roche and biosimilars.

China has become the first country in the world to approve Roche’s anti-complement C5 antibody crovalimab, the only treatment for the rare disorder paroxysmal nocturnal haemoglobinuria (PNH) that can be given by subcutaneous injection.

AstraZeneca has claimed its first regulatory approval for oral Factor D inhibitor danicopan, getting a green light in Japan for the drug as Voydeya for the treatment of paroxysmal nocturnal haemoglobinuria (PNH).

The week after Novartis secured a foothold in the paroxysmal nocturnal hemoglobinuria (PNH) market, AstraZeneca has released new data from its own attempt to find fresh ways of tackling this rare blood disease.

Novartis has announced that the US Food and Drug Administration (FDA) has given approval to Fabhalta (iptacopan) as the first oral monotherapy to treat adults suffering with paroxysmal nocturnal hemoglobinuria (PNH).

Astrazeneca has the dominant complement inhibitor franchise via the acquisition of Alexion and its intravenous therapies Soliris and Ultomiris. But that position could soon come under fire from Novartis’s oral contender iptacopan.

Astra has oral projects of its own, although these are either way behind or focused on relatively small niches in paroxysmal nocturnal haemoglobinuria (PNH), their first indication. But Anita Hill, vice-president of global medical affairs at the Alexion division, believes that there is a good reason not to write off Soliris and Ultomiris just yet.

Novartis has announced positive data from a Phase III trial investigating iptacopan in patients with paroxysmal nocturnal haemoglobinuria (PNH). The study met its primary endpoint and demonstrated clinically meaningful benefits across secondary endpoints.