Drugs
-
Naldemedine [Symproic® (Japan; USA); Rizmoic® (EU)], an orally available peripherally acting μ-opioid receptor antagonist (PAMORA), is approved in several countries for the treatment of opioid-induced constipation. In phase III trials, naldemedine was more effective than placebo at increasing the frequency of bowel movements in patients with constipation induced by opioid treatment for cancer pain or chronic non-cancer pain. Naldemedine was also associated with improvements in patient-rated constipation-related symptoms and quality of life. ⋯ Because naldemedine specifically targets opioid receptors in the gastrointestinal (GI) tract and does not cross the blood-brain barrier, it does not cause opioid withdrawal symptoms or interfere with centrally mediated opioid analgesia. Consistent with its mechanism of action, the most commonly reported adverse events were GI in nature. In conclusion, current data indicate that naldemedine is an effective and generally well-tolerated treatment option for opioid-induced constipation in patients with cancer pain or chronic non-cancer pain, with the convenience of once-daily oral dosing.
-
Onasemnogene abeparvovec (onasemnogene abeparvovec-xioi; formerly AVXS-101; ZOLGENSMA®) is an adeno-associated viral vector-based gene therapy designed to deliver a functional copy of the human survival motor neuron (SMN) gene to the motor neuron cells of patients with spinal muscular atrophy (SMA). It has been developed by AveXis, a Novartis company, and was approved in May 2019 in the USA for the treatment of paediatric patients aged < 2 years with SMA and bi-allelic mutations in the SMN1 gene (the primary gene encoding survival motor neuron protein). ⋯ Regulatory assessments for this formulation of onasemnogene abeparvovec are underway in the EU and Japan; an intrathecal formulation is currently undergoing clinical development in the USA. This article summarizes the milestones in the development of onasemnogene abeparvovec leading to this first approval for the treatment of paediatric patients aged < 2 years with SMA and bi-allelic mutations in SMN1.