FDA approves Bkemv, first Soliris biosimilar, to treat PNH

Considered interchangeable, Bkemv may be given at pharmacy's discretion

Andrea Lobo, PhD avatar

by Andrea Lobo, PhD |

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The U.S. Food and Drug Administration (FDA) has approved Bkemv (eculizumab-aeeb), the first interchangeable biosimilar of Soliris (eculizumab), to treat people with paroxysmal nocturnal hemoglobinuria (PNH).

Soliris is an intravenous (into-the-vein) antibody-based therapy that is approved to reduce hemolysis, or red blood cell destruction, in PNH patients. As a biosimilar, Bkemv, developed by Amgen, was found to be highly similar to Soliris — its reference medication — and equivalent in regard to its quality, safety, and efficacy.

Since biosimilars require much less research and development than a reference medication, they are usually available at lower prices, helping with patient access to treatment.

An interchangeable biosimilar may be substituted for its reference therapy without a request to the prescribing healthcare provider. As with generic medications, this substitution may occur at the pharmacy if state regulations allow such changes.

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“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,” Sarah Yim, MD, director of the Office of Therapeutic Biologics and Biosimilars in the FDA’s Center for Drug Evaluation and Research, said in an agency press release.

A rare disease, PNH usually is caused by mutations in the PIGA gene. This gene provides instructions to produce a protein that’s required for the production of a molecule that works to attach other proteins to the surface of cells, including CD55 and CD59, proteins found in red blood cells.

The lack of these proteins, which normally help to identify red blood cells to the immune system as healthy body parts, leads to the activation of the complement system, a part of the immune system, causing hemolysis.

Eculizumab, the active ingredient in both Soliris and Bkemv, works by binding to a complement protein called C5, preventing its cleavage and blocking complement activation. As such, the therapy can reduce hemolysis and prevent anemia, a hallmark symptom of PNH.

The Phase 3 DAHLIA trial (NCT03818607) compared the safety and efficacy of Bkemv to that of Soliris in 42 adults with PNH, enrolled at several sites in the U.S. and Europe. Patients randomly were assigned to either Bkemv treatment for one year followed by Soliris for six months, or vice versa. Both therapies were given every two weeks by intravenous injection at a 900 mg dose.

It met its primary goal of showing no clinically meaningful differences between Bkemv and Soliris regarding their ability to control intravascular hemolysis, or red blood cell destruction taking place inside blood vessels.

Bkemv also carries a boxed warning of a meningococcal infection risk

Bkemv is expected to have the same side effects as Soliris, which can include headache, the common cold, back pain, and nausea.

Bkemv’s prescribing information also comes with a boxed warning, which states that products containing eculizumab may increase the risk of serious or life-threatening meningococcal infections caused by the bacteria Neisseria meningitidis.

At least two weeks before starting treatment with Bkemv or Soliris, patients should have completed or updated their meningococcal vaccination scheme, unless the risks of delaying treatment outweigh the risks of a potentially serious infection. Additionally, they should be monitored for early signs and symptoms of meningococcal infections during treatment, and be evaluated immediately in case an infection is suspected.

Like Soliris, Bkemv is available only through a restricted program called a Risk Evaluation and Mitigation Strategy (REMS). This program is required by the FDA for certain medications with serious safety concerns to help ensure that treatment benefits outweigh potential risks. Under a REMS program, only certified healthcare professionals can prescribe Bkemv and the medication can be dispensed only at certified pharmacies and other healthcare settings.

In addition to PNH, Bkemv is approved to treat a rare disease called atypical hemolytic uremic syndrome (aHUS), helping to prevent blood clots from forming in small blood vessels due to the overactivation of the complement system.