The European Union (EU)'s Committee for Orphan Medicinal Products (COMP) has released a tranche of positive opinions for orphan-drug designations for 17 medicines in Europe.
IHS Life Sciences perspective | |
Implications | The EU's COMP published positive opinions for the inclusion of new medicinal products in the European registry of orphan medicines on 24 February 2016. |
Outlook | The COMP recommendations are subject to a final approval process by the European Commission. The commission is expected to publish decisions within 30 days of receiving the COMP's designation. Inclusion on the orphan-drug registry enables manufacturers to secure 10-year exclusivity in the EU upon marketing-authorisation approval, in addition to development incentives. |
The European Union (EU)'s Committee for Orphan Medicinal Products (COMP) has issued 17 recommendations for orphan-medicine designations in Europe (the full COMP document is available to view here). No negative COMP decisions were published; however, the COMP noted that five applications for orphan-medicine-product designation were withdrawn.
COMP orphan-medicine recommendations | ||
Endorsements adopted at second session | Company / organisation | Indication |
Acalabrutinib | Acerta Pharma (Netherlands/US) | Chronic lymphocytic leukaemia / small lymphocytic lymphoma |
Allogeneic Epstein-Barr virus specific cytotoxic T lymphocytes | Wainwright Associates (UK) | Treatment of post-transplant lymphoproliferative disorder |
Fenretinide | Clinipace (US) | Cutaneous T-cell lymphoma |
Glucopyranosyl lipid A stable emulsion and recombinant New York oesophageal squamous-cell carcinoma-1 | Pharm Research Associates (UK) | Soft-tissue sarcoma |
Sindbis virus-envelope pseudotyped lentiviral vector-encoding New York oesophageal squamous-cell carcinoma-1 | Pharm Research Associates (UK) | Soft-tissue sarcoma |
Florilglutamic acid | Piramal Imaging GmbH (Germany) | Diagnosis of hepatocellular carcinoma / diagnosis of glioma |
Fosbretabulin tromethamine | Diamond BioPharm (UK) | Gastro-entero-pancreatic neuroendocrine tumours |
Synthetic double-stranded siRNA oligonucleotide directed against hydroxyacid oxidase 1 mRNA and covalently linked to a ligand containing three N-acetylgalactosamine residues | Alnylam (UK) | Primary hyperoxaluria |
Ubenimex | Eiger Biopharmaceuticals (US) | Pulmonary arterial hypertension |
Endorsements adopted at first session | Company / organisation | Indication |
Acalabrutinib | Acerta Pharma (US) | Lymphoplasmacytic lymphoma |
Acalabrutinib | Acerta Pharma (US) | Mantle-cell lymphoma |
Adeno-associated viral vector serotype 5 containing a B-domain-deleted variant of human coagulation factor VIII gene | BioMarin (US) | Haemophilia A |
Adeno-associated viral vector serotype 8 encoding human ornithine transcarbamylase | Pharma Gateway AB (Sweden) | Ornithine transcarbamylase deficiency |
Diaspirin cross-linked haemoglobin | New B Innovation (UK) | Oesophageal cancer |
Exenatide | Alan Boyd Consultants (UK) | Idiopathic intracranial hypertension |
N-acetyl-D-mannosamine monohydrate | Escala Therapeutics (US) | GNE myopathy |
Source: COMP | ||
Outlook and implications
The coveted orphan-drug designation targets medicinal products that are in development for the treatment of rare diseases affecting fewer than 5 per 10,000 persons living in the EU. The designation is highly positive for the applicant companies, as it enables manufacturers to obtain a 10-year period of patent protection in the EU, provided that marketing authorisation is subsequently granted. In addition, the period of market exclusivity can be extended by a further two years in the case of paediatric medicine approvals. Development incentives – including protocol assistance, scientific advice, and reduced regulatory fees – are also linked to the designation.
The COMP recommendation moves the companies concerned a step closer to the European Commission's final approval for orphan-drug status, which is usually confirmed within one month of issuance of the COMP opinion.
Among the positive recommendations granted by the COMP, of particular significance were the endorsements received by Acerta Pharma's Acalabrutinib for multiple orphan-drug designations for the treatment of chronic lymphocytic leukaemia, small lymphocytic lymphoma, lymphoplasmacytic lymphoma, and mantle-cell lymphoma. The recommendation comes in the wake of AstraZeneca (UK)'s completed acquisition of a majority stake of 55% in Acerta Pharma in February. The transaction entailed an upfront payment of USD2.5 billion, and a commitment by the UK firm to make a further unconditional payment of USD1.5 billion upon the first approval of the investigational, irreversible oral Bruton's tyrosine-kinase (BTK) inhibitor, acalabrutinib (ACP-196) in the United States, or by the end of 2018. In return, AstraZeneca secured rights to acalabrutinib – which is currently undergoing Phase III development for B-cell blood cancers – as well as Phase I/II clinical trials in multiple solid tumours. Assuming that regulatory approvals proceed favourably for Acerta / AstraZeneca, the candidate acalabrutinib holds the potential to generate peak global sales of USD7.5 billion. As part of the agreement, AstraZeneca also gained an option to purchase the remaining shares in the US company. Clinical-trial data on acalabrutinib have been encouraging: Phase I/II data for the BTK inhibitor demonstrated a 95% response rate in patients with relapsed chronic lymphocytic leukaemia, and an overall response rate of 100% in the difficult-to-treat 17p deletion patients. Acalabrutinib is additionally being investigated in combination with immunotherapy or chemotherapy in solid tumours. AstraZeneca is targeting initial regulatory submissions in the second half of 2016 for specific types of haematological malignancies.
Elsewhere, US-based firm Retrophin announced that it had received an orphan-drug designation from the European Commission for RE-024, a novel phosphopantothenate-replacement therapy in the treatment of the extremely rare, life-threatening genetic disorder pantothenate kinase-associated neurodegeneration (PKAN). The disease is estimated to affect just three persons per million globally (equivalent to a potential patient pool of about 5,000 individuals worldwide). The orally-administered therapy is currently undergoing early-stage development.