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With FDA approval, Apellis can challenge AstraZeneca in rare blood disorder

Apellis Pharmaceuticals has FDA approval for its first product, setting the stage for a head-to-head competition against a pair of blockbuster therapies in line to soon join the portfolio of pharmaceutical giant AstraZeneca.

The Apellis drug, pegcetacoplan, is a treatment for paroxysmal nocturnal hemoglobinuria (PNH), a rare blood disorder in which part of a patient’s immune system called the complement system attacks red blood cells, leading to blood clots and organ damage. PNH is a chronic condition and the resulting damage to red blood cells requires PNH patients to undergo frequent blood transfusions. The disease affects an estimated 15,000 people worldwide, according to Apellis.

Waltham, Massachusetts-based Apellis designed its peptide drug to target and block a complement system protein called C3. It’s the first approved drug that targets this protein. The Apellis drug, a twice weekly infusion, will be marketed under the name “Empaveli.”

The FDA based its approval on the results of a Phase 3 study that tested Empaveli head to head against Soliris, an Alexion Pharmaceuticals drug already approved to treat PNH. In the study, the Apellis drug beat Soliris in increasing hemoglobin levels after 16 weeks of treatment, which was the main goal. The hemoglobin levels achieved at 16 weeks were sustained through 48 weeks. The Apellis drug also was also no worse than Soliris in the secondary goal of avoiding transfusions. According to the study results, which were published in March in the New England Journal of Medicine, 85% of patients treated with Empaveli were transfusion free over the course of 16 weeks compared to 15% of patients treated with Soliris.

The most common side effects observed in the study included injection site reactions, infections, diarrhea, abdominal pain, respiratory tract infection, viral infection, and fatigue. Infection was the most common serious adverse event reported in the study, and the drug’s label contains a black box warning that points out infection risks from encapsulated bacteria. As a condition of regulatory approval, the FDA is requiring Apellis to develop a plan that informs clinicians and patients about these risks and manages them.

Alexion’s Soliris is among the most expensive medicines, costing more than $678,000 annually, according to GoodRx. That drug, which is approved for several other rare diseases in addition to PNH, accounted for more than $1 billion of Alexion’s 2020 revenue. Boston-based Alexion has been transitioning Soliris patients to that drug’s successor, Ultomiris. Both drugs target the C5 protein, but Ultomiris is longer acting and is infused every eight weeks compared to every two weeks for Soliris. Sales of Ultomiris were just over $1 billion last year.

FDA approval of Empaveli clears the way for Apellis to try and steal market share from both Alexion drugs. The regulatory decision covers adult patients who have never received treatment, as well as those switching from either Soliris or Ultomiris. Apellis contends its drug can help patients for whom a C5 inhibitor is insufficient. In an investor presentation, the company pointed to data showing that of 1,500 patients treated by a C5 inhibitor, one third still needed transfusion to boost falling hemoglobin levels while another third of patients experienced symptoms like fatigue due to low hemoglobin. With that competition in mind, Apellis said the average wholesale price for its drug, before any discounts or rebates, will be $458,000. That annual cost is on par with Ultomiris and at a discount to the pricier Soliris.

Those Alexion drugs are set to become part of a new rare disease drug unit within AstraZeneca. Late last year, the British pharmaceutical company agreed to acquire Alexion in a $39 billion deal. The Federal Trade Commission cleared the acquisition in April. Last week, shareholders of both AstraZeneca and Alexion voted in favor of the buyout. The pharma giant said it continues to expect that the transaction will close in the third quarter of this year.

Photo: virusowy, Getty Images

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