- Orphan drugs accounted for nearly half of the Committee for Medicinal Products for Human Use (CHMP)'s most recent round of approval recommendations.
- The committee, tasked with evaluating whether medicines should progress into the E.U. market, gave positive opinions on 11 treatments during its July meeting, five of which had orphan drug designation. That subset included Pfizer and Merck KGaA's Bavencio (avelumab), Novartis' Rydapt (midostaurin), Advanced Accelerator Applications' Lutathera (lutetium Lu 177 dotatate), Santen Pharmaceuticals' Verkazia (ciclosporin) and Ipsen Pharma's Xermelo (telotristat ethyl).
- The good news extended outside the realm of rare diseases to Sanofi and Regeneron's eczema drug Dupixent (dupilumab), Roche's Tecentriq (atezolizumab) for a pair of cancer indications, and handful of other medicines. The CHMP also rejected two drugs: Vanda Pharmaceuticals' schizophrenia therapy Fanaptum (iloperidone) and Nektar Therapeutics' topoisomerase 1-inhibitor Onzeald (etirinotecan pegol).
The development of orphan drugs is increasing around the world with both the U.S. and EU offering marketing incentives to drugmakers. While the U.S. offers a wider slate of incentives, drugmakers can still get 10 years of market exclusivity for launching an orphan drug on the EU market.
Due to promise of these medicines, orphan drugs are expected to account for about $209 billion in worldwide drug sales by 2020, according to a recent report by EvaluatePharma.
Looking at the CHMP's July orphan drug approval recommendations, four fight cancers or tumor-related conditions, while the fifth works on a rare eye condition called vernal keratoconjunctivitis in children.
Bavencio is part of the first group. The Pfizer and Merck KGaA drug is an anti-PD-L1 immunotherapy indicated for locally advanced or metastatic urothelial carcinoma and metastatic Merkel cell carcinoma (MCC).
In its opinion, the EMA committee said that "the benefits with Bavencio are its ability to show tumour responses in patients whether they have or not been treated with chemotherapy. In many patients, the response is of prolonged duration."
As for Novartis' Rydapt, European regulators were impressed with the overall survival rates seen across the majority of patients treated with the drug. The CHMP recommended the treatment's approval in four different indications: acute myeloid leukaemia, aggressive systemic mastocytosis, systemic mastocytosis with associated haematological neoplasm and mast cell leukaemia.
Lutathera and Xermelo are tumor-focused therapies.
The former is for patients with gastroenteropancreatic neuroendocrine tumors, and received a positive opinion based on Phase 3 data showing it bested the somatostatin receptor agonist octreotide LAR at improving progression free survival. The latter was given a CHMP's thumbs up as a treatment, in combination with a somatostatin analogue, for the diarrhea that patients with carcinoid syndrome often experience.
Lastly, Verkazia aims to help children and adolescents with severe vernal keratoconjunctivitis, a rare eye condition that can result in symptoms ranging from itchy eyes to photophobia.
"The benefits of Verkazia are its ability to improve ocular surface damage and reduce symptoms of severe vernal keratoconjunctivitis in children and adolescent patients," the CHMP said in its opinion. "The most common side effects are eye pain (11%) and eye pruritus (9%) which usually occur at the time of instilling the eye drops."
Once the CHMP gives its scientific opinion on a product, the European Commission —taking into account the EMA opinion —grants marketing authorization for the product.