AstraZeneca's Oral Drug Danicopan Receives CHMP Backing for PNH-Related Anaemia Treatment

Key Insights

• The EMA's CHMP has recommended approval of AstraZeneca's danicopan as an add-on therapy for patients with paroxysmal nocturnal haemoglobinuria (PNH) who experience residual anaemia despite C5 inhibitor treatment.
• Clinical trial data from the ALPHA study demonstrated significant improvements in haemoglobin and reticulocyte count levels when danicopan was combined with existing C5 inhibitors Soliris or Ultomiris.
• If approved, danicopan will offer the first oral alternative to current injectable treatments for PNH patients with residual anaemia, affecting approximately 10-20% of those receiving C5 inhibitor therapy.

AstraZeneca has achieved a significant milestone in its pursuit of expanding treatment options for paroxysmal nocturnal haemoglobinuria (PNH) patients, as the European Medicines Agency's Committee for Medicinal Products for Human Use (CHMP) recommends approval of its oral Factor D inhibitor, danicopan.

The drug, already marketed as Voydeya in Japan, is specifically targeted as an add-on therapy for PNH patients experiencing residual haemolytic anaemia despite treatment with C5 inhibitors such as Soliris (eculizumab) or Ultomiris (ravulizumab).

Addressing an Unmet Medical Need

PNH, a rare and life-threatening blood disorder, continues to present treatment challenges, with approximately 10-20% of patients maintaining low red blood cell levels despite C5 inhibitor therapy. Currently, these patients can only access Apellis' Aspaveli (pegcetacoplan), administered through subcutaneous infusion. Danicopan's oral formulation represents a potential breakthrough in treatment convenience and accessibility.

Clinical Evidence Supporting Approval

The CHMP's positive opinion is founded on compelling data from the ALPHA study. The trial demonstrated that combining danicopan with either Soliris or Ultomiris resulted in marked improvements in key clinical markers, including:

• Significant elevation in mean haemoglobin levels
• Improved absolute reticulocyte count (ARC) levels compared to C5 inhibitor monotherapy in patients with residual anaemia.

Strategic Implications and Future Prospects

While market analysts project modest sales for danicopan in the PNH indication, the drug serves a crucial role in AstraZeneca's strategy to protect its C5 inhibitor franchise. This becomes particularly relevant as new competitors enter the market, including Roche's crovalimab, which recently secured approval in China.

Beyond PNH, AstraZeneca is exploring danicopan's potential in geographic atrophy, a leading cause of vision loss, potentially opening up a substantially larger market opportunity for the drug.