AstraZeneca gives rare diseases new challenge

United Kingdom-based global pharmaceutical company AstraZeneca has integrated more than 10 molecular products worldwide in the rare diseases field and is promoting more than 20 clinical research programs, said an executive of the company.

The company will continue to introduce new product portfolios over the upcoming five years after announcing joining the rare diseases realm in 2021, said Hu Yiqing, vice-president of AstraZeneca China and head of its rare diseases business unit, in an interview with China Daily ahead of the International Rare Diseases Day, which fell on Tuesday this year.

Areas of focus will include hematology, nephrology, neurology, metabolic disorders and ophthalmology, she said.

"In addition to the acquisition of Alexion, which specializes in rare disease therapies, in 2020, AstraZeneca announced the acquisition of LogicBio Therapeutics, which had a leading position in the development of gene delivery and gene editing and was committed to tackling various rare and serious diseases afflicting children and adults," said Hu.

"Therefore, the company will expand its layout of rare diseases at the level of genomics," she said.

In terms of research and development in the company's rare diseases therapy catalog, Hu said China and the rest of the world are developing at the same time.

"In addition to the introduction of innovative medicines that have entered the markets overseas to China, all the therapies that are under research progress will be promoted into clinical trials in China simultaneously," she said.

In November, China approved the first rare disease therapy from AstraZeneca, bringing innovative treatment options to paroxysmal nocturnal hemoglobinuria and atypical hemolytic-uremic syndrome, two diseases related to kidney and blood vessel malfunctions in adults and children.

The company is pushing forward the therapy's approval for other new indications and the approval for another medicine to treat a rare neurofibroma, a neurological condition, to benefit more patients, said Hu.

Data from market research firm Frost & Sullivan showed that the size of the rare disease treatment market in China is expected to surge from $1.3 billion in 2020 to $25.9 billion in 2030 with a compound annual growth rate of 34.5 percent. Experts believe that it is a result of the actual needs of the patients.

The country has unveiled favorable measures to streamline a new medicine market used in the rare disease field. Some therapies with clinical data overseas can register without clinical data domestically to accelerate foreign innovative drugs being brought into the country, according to experts.

Besides acceleration in bringing new therapies, Hu said the company will also contribute to building a more complete diagnosis and treatment system regarding rare diseases.

The country has established a collaboration network of rare diseases among hospitals nationwide and 60 of them were designated as medical centers. Hu said AstraZeneca will help strengthen multidisciplinary treatment capability at the medical centers as rare diseases are usually diagnosed jointly by doctors from various backgrounds.

"These medical centers will later expand their practice and standardize diagnosis and treatment of relevant diseases at other medical institutions. Also, the hospitals will build transfer mechanisms for high-risk patients," said Hu.