Multiple orphan medications get nod from EMA
medwireNews: Lutetium-177 (177Lu)-oxodotreotide is one of four medications with an orphan designation to receive a positive opinion from the European Medicines Agency (EMA) in the most recent round of assessments, the others being avelumab, midostaurin, and telotristat ethyl.
The radiopharmaceutical product 177Lu-oxodotreotide is indicated for adult patients with inoperable or metastatic, well differentiated gastroenteropancreatic neuroendocrine tumors expressing somatostatin receptors.
The decision is based on results showing improved progression-free survival with 177Lu-oxodotreotide over the somatostatin receptor agonist octreotide LAR in this patient population.
The committee has recommended granting a marketing authorization to avelumab for the treatment of adults with metastatic Merkel cell carcinoma. The programmed cell death ligand 1 inhibitor is to be given as a single agent and will be available as a concentrated 20 mg/mL solution for infusion.
The decision summary highlights the ability of avelumab to elicit tumor responses – frequently durable – regardless of whether patients have received prior chemotherapy or not.
Midostaurin, a multitargeted receptor tyrosine kinase inhibitor, was designated as an orphan medicinal product in 2004 and has now received a positive opinion from the EMA.
The drug is intended for adult patients with a new diagnosis of FLT3 mutation-positive acute myeloid leukemia, to be given alongside standard daunorubicin and cytarabine induction and high-dose cytarabine consolidation chemotherapy.
As per the decision, patients with aggressive systemic mastocytosis, systemic mastocytosis with associated hematologic neoplasm, or mast cell leukemia can also receive midostaurin monotherapy.
The EMA has adopted a positive opinion with regards to telotristat ethyl for patients with carcinoid syndrome-related diarrhea. It is to be given alongside somatostatin analog (SSA) therapy to patients whose symptoms are inadequately controlled by SSAs.
The agent, which inhibits L-tryptophan hydroxylases, will be available as film-coated tablets of 250 mg.
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