FDA approves Bkemv, biosimilar of Soliris, to treat aHUS

As an interchangeable medication, pharmacy may decide to dispense Bkemv

Margarida Maia, PhD avatar

by Margarida Maia, PhD |

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The U.S. Food and Drug Administration (FDA) has approved Bkemv (eculizumab-aeeb), the first interchangeable biosimilar to Soliris (eculizumab), to treat people with atypical hemolytic uremic syndrome (aHUS).

A biosimilar is a medication highly similar to a reference biologic — a medication made from natural, living sources like bacteria — already approved by the FDA. It is expected to be as safe and work as well over the course of treatment as its reference biologic product, Soliris in this case, but it is expected to cost less.

In the U.S., a clinically equivalent biosimilar with an “interchangeable” designation can be substituted for its reference biologic at the pharmacy without consulting the prescribing doctor, state pharmacy laws permitting.

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Bkemv, by Amgen, also was approved to treat paroxysmal nocturnal hemoglobinuria (PNH), another rare disease that, like aHUS, occurs when the complement system — part of the immune system — becomes overly active, causing red blood cells to break down.

“Many rare conditions are life-threatening, and many do not have treatments,” Sarah Yim, who directs the Office of Therapeutic Biologics and Biosimilars in the FDA’s Center for Drug Evaluation and Research, said in an agency press release.

“The FDA is committed to help facilitate the development of safe and effective interchangeable biosimilar treatments that can expand access for individuals with rare diseases whose current treatment options are limited,” Yim added.

Bekemv was approved in Great Britain to treat adults and children with PNH in October 2022, and it was approved in the European Union for the same indication in April 2023.

Like Alexion’s Soliris, Bkemv works by binding to C5, a complement protein, preventing the complement system from becoming overly active and breaking down red blood cells, thereby helping to control aHUS symptoms.

Inhibiting complement activity in people with aHUS is expected to ease thrombotic microangiopathy, which occurs when clots form in small blood vessels, blocking blood flow to tissues and damaging organs, particularly the kidneys.

In an Amgen-funded study involving 217 healthy men, a single 300 mg intravenous (into-the-vein) infusion of Bkemv — then known as ABP 959 — matched Soliris in terms of how fast it was taken up into the bloodstream and how long it stayed in the body.

It also was seen to be as safe and to inhibit complement activity as well as Soliris. None of the participants developed neutralizing antibodies against Bkemv or Soliris, which could prevent either medication from working as expected.

Like Soliris, Bkemv carries a boxed warning concerning eculizumab

In a Phase 3 clinical study called DAHLIA (NCT03818607), 42 adults with PNH were assigned to receive 900 mg of Bkemv or Soliris, given every 14 days, for two periods of treatment in a random order. Patients receiving Bkemv in the first period of treatment (lasting around one year) then switched to Soliris in the second treatment period (lasting about six months), and vice versa.

Bkemv showed similar clinical efficacy to Soliris, as measured by the levels of lactate dehydrogenase, an enzyme released from red blood cells when they break down.

While Bkemv has not been tested in people with aHUS, researchers found that it can block the C5 complement protein in a way similar to Soliris in a lab model of simulated blood from the disease, supporting its use in aHUS to ease thrombotic microangiopathy.

Like Soliris, Bkemv’s label carries a boxed warning noting that products containing eculizumab can increase risk of serious and life-threatening meningococcal infection. Patients must complete meningococcal vaccination before starting treatment, be monitored for infection signs, and seek care if symptoms appear.

Bkemv will be available only through a restricted program, called a Risk Evaluation and Mitigation Strategy (REMS), which the FDA requires for certain medications with serious safety concerns to help ensure treatment benefits outweigh potential risks.