AstraZeneca’s Ultomiris Approved for Children and Adolescents with PNH

The European Commission (EC) has approved global pharmaceutical company AstraZeneca PLC’s (AZN) Ultomiris (ravulizumab) to treat adolescents and children suffering from an ultra-rare and severe blood disorder called paroxysmal nocturnal haemoglobinuria (PNH). The disorder is characterized by the destruction of red blood cells causing thrombosis (blood clots) that can lead to organ damage and premature death.

AstraZeneca is engaged in the production and sale of medicines for the treatment of cancer, respiratory and inflammation, oncology, neuroscience, cardiovascular, gastrointestinal and other infectious diseases. (See AstraZeneca stock chart on TipRanks)

The approval was based on interim results from the Phase III clinical trial that demonstrated the safety and efficacy of Ultomiris in children and adolescents with PNH. The European Medicines Agency’s Committee for Medicinal Products for Human Use recommended the drug in July 2021.

The Managing Director at King’s College Hospital, London, Austin Kulasekararaj, said, “PNH is a devastating disease, and Ultomiris provides advancement for pediatric patients in the EU with an established safety and efficacy profile. By requiring fewer infusions each year than Soliris, Ultomiris may reduce the need for these young patients to miss school to receive treatment.”

The EC approved Ultomiris for the treatment of adults with PNH as well as for the treatment of adults and children with atypical hemolytic uremic syndrome (aHUS) in 2019. The U.S. Food and Drug Administration (FDA) approved the drug for children and adolescents with PNH in June 2021.

Last month, Bank of America Securities analyst Sachin Jain reiterated a Buy rating on the stock with a price target of $69.4 (18% upside potential).

The analyst said, “Due to lack of success of the only other biologic indicated for SLE Benlysta, market expectations for anifrolumab are low, in our view. However, given the potential size of the market, and potential points of differentiation of anifrolumab versus Benlysta, we believe anifrolumab could offer upside to market expectations.”

Overall, the stock has a Strong Buy consensus rating based on 3 unanimous Buys. The average AstraZeneca price target of $68.95 implies 17.2% upside potential. Shares of the company have gained 22.5% over the past six months.

According to TipRanks’ Smart Score rating system, AstraZeneca scores an 8 out of 10, suggesting that the stock is likely to outperform market averages.