AstraZeneca’s Rare Disease Pipeline Notches Another FDA Approval

An AstraZeneca drug acquired in a $930 million deal has received FDA approval as a treatment for a rare blood disorder.

In this disease, paroxysmal nocturnal hemoglobinuria (PNH), a part of the immune system called the complement system destroys red blood cells. PNH can be treated with the blockbuster AstraZeneca medications Soliris and Ultomiris, both of which are standard of care therapies.

The new AstraZeneca drug, danicopan, brand name Voydeya, is approved as an add-on to standard PNH drugs for patients who still experience extravascular hemolysis, the breakdown of red blood cells outside of blood vessels. Soliris and Ultomiris are antibody drugs designed to block the complement system protein C5. These drugs are administered as intravenous infusions. Voydeya is a three-times-a-day oral pill designed to block Factor D, a protein that plays a role in amplifying the complement system response. According to AstraZeneca, about 10% to 20% of patients with PNH experience clinically significant extravascular hemolysis while being treated with a C5 inhibitor.

In Phase 3 testing, adding Voydeya to Soliris or Ultomiris met the main goal of showing a change in hemoglobin levels measured at week 12. The drug also met secondary goals that included transfusion avoidance and a change in score according to a rating assessment that measures fatigue and its impact on daily activities and function. The study results showed Voydeya was generally well tolerated. The most common adverse effects reported included headache, nausea, joint stiffness, and diarrhea. The Phase 3 results were published in The Lancet Haematology in late 2023.

Voydeya was developed within Alexion, AstraZeneca Rare Disease, the pharma giant’s rare disease division created from the 2021 acquisition of Alexion Pharmaceuticals for $39 billion. As a standalone company, Alexion had already commercialized both Soliris and Ultomiris, whose approvals span several complement system disorders. Soliris, which starts with weekly dosing for the first four weeks and then maintenance dosing every other week, accounted for about $3.1 billion in revenue last year. Ultomiris, designed for dosing every eight weeks, generated $2.9 billion in sales last year.

Alexion’s 2019 acquisition of Achillion Pharmaceuticals brought the small molecule that would become Voydeya. At the time of the deal, Achillion had already advanced that PNH drug candidate to mid-stage clinical testing in combination with Soliris.

Japan was the first country to approve Voydeya, a regulatory decision handed out in January. Regulatory reviews are underway in Europe and other markets around the world. AstraZeneca is also exploring potential expansion of the drug’s label. A Phase 2 clinical trial is testing the drug as a monotherapy for geographic atrophy, an advanced form of age-related macular degeneration associated with excessive complement system activity.

Not all bets with Alexion’s drugs have paid off. A Phase 3 test of Ultomiris in amyotrophic lateral sclerosis failed in 2021. But AstraZeneca is pursuing development of other rare disease drug candidates that came from Alexion. The pharma giant has also looked outside the company for opportunities to expand its scope in rare disease. Last month, AstraZeneca reached an agreement to buy Amolyt Pharma for $800 million up front. Amolyt’s lead program is in Phase 3 testing for the hypoparathyroidism, a disease that currently has only one FDA-approved treatment.

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