US approval of UCB’s Zilbrysq provides a second self-administration option for AChR antibody-positive patients with the rare disease.
The US Food and Drug Administration (FDA) has approved UCB’s Zilbrysq (zilucoplan) as the first macrocyclic peptide C5 inhibitor for self-administration once-daily via a subcutaneous autoinjector for adults with generalised myasthenia gravis (gMG).
The treatment provides an additional, more convenient option to a prefilled syringe (PFS) for patients who are anti-acetylcholine receptor (AChR) antibody positive.
Donatello Crocetta, Global Head of Medical Affairs and Chief Medical Officer at UCB said: “For a chronic condition like gMG, having different administration options allows patients to choose the approach that best fits their individual preferences and routines.
“From a medical standpoint, it was essential to confirm that this added administrative option didn’t come at the expense of safety and efficacy. The data supporting the autoinjector’s complete dose delivery show that this option continues to offer effective treatment for patients.”
The data supporting the autoinjector’s complete dose delivery show that this option continues to offer effective treatment for patients”
Donatello Crocetta, Global Head of Medical Affairs and Chief Medical Officer at UCB.
FDA’s approval is supported by Phase I data showing bioequivalence of Zilbrysq delivered via an autoinjector, compared with a prefilled syringe in healthy volunteers. Additionally, a Phase III study found that complete dose delivery was achieved for 99.8 percent of autoinjector administrations.
UCB’s gMG portfolio also includes Rystiggo (rozanolixizumab-noli), a subcutaneously infused monoclonal antibody that targets the neonatal Fc receptor (FcRn).
February saw Amgen gain EU approval of Uplizna (inebilizumab), as a first-in-class therapeutic approach to manage generalised myasthenia gravis.
More recently, one of two notable recent developments in the rare disease therapeutic space is Merck KGaA’s $1.3 billion acquisition of siRNA specialist Silence Therapeutics.
Additionally, the FDA has proposed a new framework for rare disease drug development. By prioritising the needs of patients, the recommendations aim to match regulatory requirements to the realities of small patient populations.
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