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17-08-2017 | Oncology | News | Article

approvalsWatch

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.

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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.

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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.

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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.

By Shreeya Nanda

medwireNews is an independent medical news service provided by Springer Healthcare. © 2017 Springer Healthcare part of the Springer Nature group

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