CHMP recommends EU approval of Voydeya as add-on PNH treatment
Therapy under review in US, approved in Japan last month
A European Medicines Agency committee has recommended the approval of Voydeya (danicopan) as an add-on treatment for adults with paroxysmal nocturnal hemoglobinuria (PNH) who continue to experience anemia despite treatment with C5 inhibitors.
Regulators will use this guidance from the Committee for Medicinal Products for Human Use (CHMP) when making a final approval decision.
Last month, the oral therapy from Alexion, AstraZeneca Rare Disease earned its first regulatory approval in Japan, which covers a similar patient population as that under consideration in Europe. The therapy is also under review in the U.S.
“If approved, Voydeya may optimise care for people impacted by this burdensome condition while allowing patients to maintain disease control with an established C5 inhibitor,” Hubert Schrezenmeier, MD, professor and medical director of the Institute of Transfusion Medicine at the University of Ulm in Germany, said in a company press release.
Hemolytic anemia is considered a hallmark of PNH. It occurs when red blood cells are destroyed faster than they can be made (hemolysis), and thus, the body does not have enough of them (anemia). This is driven by the abnormal activation of the immune system’s complement cascade.
Ultomiris, Soliris standard treatment for PNH
Inhibition of the complement C5 protein — the mechanism of approved therapies Ultomiris (ravulizumab) and Soliris (eculizumab) — is a standard treatment approach for PNH. These medications are very good at controlling intravascular hemolysis (IVH), or red blood cell destruction taking place within blood vessels.
Still, about 10%-20% of patients receiving those therapies continue to experience clinically significant extravascular hemolysis (EVH), in which red blood cells are destroyed outside of blood vessels in organs such as the spleen. This can contribute to ongoing anemia that requires blood transfusions.
“C5 inhibition with Ultomiris or Soliris is the standard-of-care in PNH, proven to control IVH and reduce life-threatening thrombotic events [blood clots], yet a small portion of patients may experience clinically significant EVH,” Schrezenmeier said.
Voydeya works by blocking factor D, another complement protein that’s activated upstream of C5. It is expected to help control EVH, while not interfering with the IVH-controlling activities of Ultomiris or Soliris when used as an add-on therapy.
The CHMP’s recommendation is based largely on data from the pivotal Phase 3 ALPHA clinical trial (NCT04469465), which showed that when taken three times daily on top of Soliris or Ultomiris, Voydeya led to significantly better control of anemia compared with a placebo after three months, with effects sustained for up to about a year.
The oral therapy was also well tolerated, and its use was associated with patient-reported improvements in life quality, reductions in fatigue, and a decreased need for blood transfusions.
“Today’s positive CHMP recommendation recognises the promise of Voydeya as an add-on to standard-of-care to address signs and symptoms of clinically significant EVH for this small subset of patients,” noted Marc Dunoyer, CEO of Alexion.
Voydeya has been previously granted orphan drug designation in the U.S., European Union (EU), and Japan, as well as breakthrough therapy designation in the U.S. and priority medicines, or PRIME status in the EU. All these designations aim to speed the therapy’s clinical development and regulatory review.
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