Dive Brief:

• Novartis is preparing next year to seek approval of a potential blockbuster rare disease drug after it succeeded in a second pivotal trial.

• The Swiss drugmaker tested the medicine, iptacopan, in patients with paroxysmal nocturnal hemoglobinuria, or PNH, a disorder in which defective red blood cells are prone to premature destruction, leaving patients anemic, fatigued or suffering from blood clots. Iptacopan is designed to prevent that destruction by interfering with the body’s natural “complement” system that cleans up damaged cells.

• Two studies have now shown that iptacopan can increase levels of hemoglobin, the crucial protein in red blood cells that carries oxygen throughout the body. An earlier trial showed the drug could help patients previously treated with AstraZeneca therapies. Research announced Thursday found it also helps patients who haven’t had those treatments.

Dive Insight:

The combined results mean Novartis can seek a broad approval of iptacopan in PNH patients and potentially position the medicine as the first oral, single therapy for the disorder. The current standard-of-care treatments, AstraZeneca’s Soliris and Ultomiris, are given intravenously.

Novartis executives have high hopes for the medicine, predicting peak sales could top $3 billion. The company is also studying the drug’s use in other diseases sparked by an overactive immune system, including a range of kidney and blood disorders.

Though PNH is considered a rare disease, treatments for it command a high enough price to turn them into blockbusters. Soliris and Ultomiris together generated revenue of almost $4.3 billion in the first nine months of this year.

Researchers are presenting results from the first Phase 3 trial of iptacopan, dubbed APPLY-PNH, at a medical meeting this weekend after flagging the success for investors in October. In that study, patients receiving the AstraZeneca therapies were randomized to either continue their treatments or receive Novartis’ drug. The iptacopan-treated patients had better hemoglobin levels and less need for transfusions.

The newer trial, APPOINT-PNH, did not have a comparison arm, instead offering iptacopan to all patients twice a day. The patients had no previous treatment with complement inhibitors, including the AstraZeneca drugs. Novartis said the study found a significant number of patients achieved “clinically meaningful” improvements in hemoglobin without the need for blood transfusions after 24 weeks.

Soliris and Ultomiris are meant to calm the body’s complement system by blocking a protein known as C5. Iptacopan, by contrast, works on a protein called factor B, which Novartis says is “upstream” of C5 in the cascade of events triggered when the complement system acts to fight damaged blood cells.