AstraZeneca highlights rare diseases in $80bn revenue push
Chief executive of subsidiary Alexion says rare disease medicine is a largely untapped area
The field of rare disease medicine remains a largely untapped area open for pharmaceutical and biotech companies to develop new treatments for thousands of diseases that lack approved therapies, the head of one of the world’s largest rare disease drugmakers has said.
Marc Dunoyer, chief executive of Alexion, the US-based rare disease subsidiary of AstraZeneca, said patients living with about “95 per cent” of the “10,000 identified, specific rare diseases” do not have medicines available to them.
“[The] rare disease world is an immense field of new opportunities,” said Dunoyer, who has led Alexion since it was acquired by AstraZeneca for $39bn in 2021.
“There are about 500 rare diseases covered by modern medicines. But about 95 per cent of them are not covered . . . [S]cience these days is making enormous leaps and progress in different avenues that five, 10 years ago were not available to us,” he told the FT in an interview.
Rare diseases are illnesses, disorders or conditions that affect a small number of people, usually about one in 2,000 individuals or fewer in the general population, according to the World Health Organization.
Alexion has become a pivotal component of AstraZeneca since its acquisition by the UK’s largest pharmaceutical group. Rare disease medicine sales accounted for 16 per cent of AstraZeneca’s $58.7bn revenue last year, making it one of the company’s biggest divisions.
The field is also regarded as a growth area as AstraZeneca sets its sights on a revenue target of $80bn by 2030 and launching 20 new medicines by the same year. AstraZeneca is halfway through meeting this new drugs pledge, and two of its new offerings are rare disease medicines.
Dunoyer, who was previously AstraZeneca’s finance chief before taking the top job at Alexion, said the Boston-headquartered division has leveraged its parent company’s global reach to expand to 80 countries from the 20 it was present in during its time as an independent company.
Alexion has announced a set of positive clinical trial data in recent weeks, including that of efzimfotase alfa, a drug to treat children with hypophosphatasia (HPP), a condition that affects bone development.
Rare disease medicine sales can be contentious given how expensive treatments can be. But Dunoyer said pricing was not necessarily the biggest concern for Alexion, although he admitted it “takes time” to agree pricing with healthcare systems.
“It takes time because these are expensive medicines for very small number of patients usually . . . it takes time for the various pricing regulators to agree and accept this financial burden,” he said, but added that many do understand the merits and savings in terms of healthcare costs and managing the burden of diseases for individuals.