UCB’s FINTEPLA®▼ (fenfluramine) oral solution has been approved in the European Union (EU) for the treatment of seizures associated with Lennox-Gastaut syndrome (LGS) as an add-on therapy to other anti-epileptic medicines for patients two years of age and older.1 The approval by the European Commission (EC) was based on safety and efficacy data from a…
NICE approves eptinezumab (Vyepti) to treat episodic and chronic migraine
On 25th January 2023, the National Institute for Health and Care Excellence (NICE) announced that eptinezumab (Vyepti) has been approved for use within the NHS in England for the treatment of episodic migraine. Eptinezumab is given by infusion once every 12 weeks. It is one of a number of calcitonin gene-related peptide (CGRP) antibody drugs, which are…
NICE Recommends Reimbursement for Translarna™
Only approved treatment for the underlying cause of nonsense mutation Duchenne muscular dystrophy PTC Therapeutics, Inc. today (19/1/23) announced that the National Institute for Health and Care Excellence (NICE) has issued a Final Evaluation Document recommending Translarna™ (ataluren) for reimbursement and use across the National Health Service (NHS) in England and Wales. Translarna is the…
Rozanolixizumab BLA for the treatment of generalised myasthenia gravis filed with US FDA and designated for priority review
UCB, a global biopharmaceutical company, announced on 6th January 2023 that the US Food and Drug Administration (FDA) has accepted the company’s filing to review a Biologic License Application (BLA) for its investigational treatment rozanolixizumab, and that the Agency has granted Priority Review.1 Rozanolixizumab is a subcutaneous (SC) monoclonal antibody targeting the neonatal Fc receptor…
Adjunctive treatment of seizures associated with Lennox-Gastaut syndrome (LGS)
FINTEPLA®▼ (fenfluramine) oral solution recommended for approval in the EU UCB, a global biopharmaceutical company, announced on 19th December 2022 that FINTEPLA®▼ (fenfluramine) oral solution has been recommended for marketing authorisation in the European Union (EU) for the treatment of seizures associated with Lennox-Gastaut syndrome (LGS) as an add-on therapy to other anti-epileptic medicines for…
Ipsen at the World Congress for Neurorehabilitation
Ipsen had a robust presence at the 12th World Congress for Neurorehabilitation (WCNR) in Vienna, Austria and virtually, from 14-17 December 2022, further demonstrating the company’s long-standing partnership with the World Federation for Neurorehabilitation (WFNR) and ongoing commitment to neuroscience. In addition to showcasing the company’s latest neuroscience clinical data, Ipsen hosted a satellite symposium…
Lecanemab: Clarity AD clinical study results
Results from Eisai’s large global Phase 3 confirmatory Clarity AD clinical study of lecanemab, an investigational anti-amyloid beta (Aβ) protofibril antibody for the treatment of mild cognitive impairment (MCI) due to Alzheimer’s disease (AD) and mild AD (collectively known as early AD) with confirmed presence of amyloid pathology in the brain, were presented at the…
Zilucoplan for the treatment of generalised myasthenia gravis in adult patients
FDA acceptance of new drug application and EMA MAA validation On 14th November 2022, UCB announced that the U.S. Food and Drug Administration (FDA) has accepted for review the New Drug Application (NDA) for its investigational treatment, zilucoplan. Zilucoplan is a subcutaneous (SC), self-administered peptide inhibitor of complement component 5 (C5 inhibitor) for the treatment…
Upstaza™ gene therapy granted marketing authorisation for AADC deficiency by MHRA
PTC Therapeutics, Inc. announced on 17th November that Upstaza™ (eladocagene exuparvovec) was granted authorisation by the Medicines and Healthcare Products Regulatory Agency (MHRA) in Great Britain. Upstaza is the first and only approved disease-modifying treatment for aromatic L-amino acid decarboxylase (AADC) deficiency and the first marketed gene therapy directly infused into the brain. It is approved for patients 18…
