Drugs
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Daratumumab (Darzalex™) is a first-in-class, humanized IgG1κ monoclonal antibody that targets the CD38 epitope and was developed by Janssen Biotech and Genmab. Intravenous daratumumab was recently approved via an accelerated approval programme in the USA for patients with multiple myeloma who have received at least three prior lines of therapy including a proteasome inhibitor (PI) and an immunomodulatory agent or who are double-refractory to a PI and an immunomodulatory agent. ⋯ In a phase II trial in patients with previously treated (as described above) relapsed or refractory multiple myeloma, monotherapy with daratumumab 16 mg/kg achieved an overall response rate of approximately 30 %. This article summarizes the milestones in the development of daratumumab leading to this first approval for multiple myeloma.
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Hypophosphatasia (HPP) is a rare inheritable disease that results from loss-of-function mutations in the ALPL gene encoding tissue-nonspecific alkaline phosphatase (TNSALP). Therapeutic options for treating the underlying pathophysiology of the disease have been lacking, with the mainstay of treatment being management of symptoms and supportive care. HPP is associated with significant morbidity and mortality in paediatric patients, with mortality rates as high as 100 % in perinatal-onset HPP and 50 % in infantile-onset HPP. ⋯ In life-threatening perinatal and infantile HPP, asfotase alfa also improved overall survival. Asfotase alfa was generally well tolerated in clinical trials, with relatively few patients discontinuing treatment and most treatment-related adverse events being of mild to moderate intensity. Thus, subcutaneous asfotase alfa is a valuable emerging therapy for the treatment of bone manifestations in patients with paediatric-onset HPP.