Orphan drugs

The US Food and Drug Administration (FDA) has approved orphan drug encorafenib (Braftovi) with binimetinib (Mektovi) for adult patients with metastatic non-small cell lung cancer (NSCLC) with a BRAF V600E mutation. The FDA also approved the FoundationOne CDx (tissue) and FoundationOne Liquid CDx (plasma) as companion diagnostics for encorafenib with binimetinib. This is the second […]

Plans to overhaul the EU regulation on rare disease therapies risk undermining two decades of progress, a new report claims. The assessment, commissioned by the European Federation of Pharmaceutical Industries and Associations (EFPIA), was conducted by Dolon, a strategic consultancy specialising in rare diseases. The European Orphan Medicinal Product Regulation was introduced in 2000 to […]

The US Food and Drug Administration (FDA) has granted orphan drug designation (ODD) to Calliditas Therapeutics for the treatment of Alport syndrome with setanaxib. Based on supportive pre-clinical work, Calliditas plans to initiate a randomised, placebo-controlled Phase II clinical study in Alport syndrome with around 20 patients in the fourth quarter of 2023. Alport syndrome […]

The US Food and Drug Administration (FDA) has granted Orphan Drug Designation to 4SC’s resminostat (Kinselby) for cutaneous T-cell lymphoma (CTCL). Resminostat is an orally administered class I, IIb and IV histone deacetylase (HDAC) inhibitor that potentially offers an approach to treating different kinds of cancer. The company demonstrated that resminostat is well tolerated and can inhibit […]

The US Food and Drug Administration (FDA) has granted Orphan Drug Designation to DTx Pharma’s DTx-1252, an investigational small interfering RNA (siRNA) therapeutic for the treatment of Charcot-Marie-Tooth Disease Type 1A (CMT1A). CMT1A is a progressive, neuromuscular, autosomal-dominant disease that leads to life-long loss of muscle function and disability. “We are pleased to receive Orphan […]

The US Food and Drug Administration (FDA) has granted Orphan Drug Designation to SiSaf’s small interfering RNA (siRNA) therapeutic for skeletal disorder Autosomal Dominant Osteopetrosis Type 2 (ADO2). In addition, due to the serious manifestations of this rare disease in children, SIS-101-ADO has been granted Rare Pediatric Disease Designation for the treatment of Autosomal Dominant […]

A new strategic guide, intended to help manufacturers bring rare disease products to market, has been launched to mark Rare Disease Day (February 28). In the guide, Terebellum (a subsidiary of AscellaHealth) shares tips on how industry stakeholders can create an effective market access strategy, giving their treatment the best chance of success in a competitive […]

Spanish pharma company Ferrer and Swiss biotech Asceneuron have signed a licensing agreement giving Ferrer the rights to ASN90 in progressive supranuclear palsy (PSP), an orphan, tau-related disease with a high unmet medical need.  The terms of the agreement include an upfront fee and multiple development, regulatory and commercial milestone payments. Asceneuron is also eligible […]

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).  It has received authorisation as an add-on therapy to other anti-epileptic medicines for patients two years of age and older.  The approval was based on safety and efficacy data from a global, […]

The US Food and Drug Administration (FDA) has designated the investigation of Endogena Therapeutics’ EA-2353 for the treatment of retinitis pigmentosa (RP) as a Fast Track development programme.   Fast Track is a process designed to enable patients to benefit earlier from important new drugs for serious conditions.   EA-2353 takes a novel, small-molecule approach and selectively […]

The European Commission has granted orphan designation for Krystal Biotech’s KB407 for the treatment of cystic fibrosis (CF).  KB407 is an investigational, redosable gene therapy designed to correct the underlying cause of cystic fibrosis by delivering two copies of the CFTR gene directly to the airway epithelial cells when delivered via a nebuliser.   By inducing […]

An investigational one-time gene therapy for the treatment of adults with severe haemophilia A has been proven to be effective for more than three years from treatment.  BioMarin Pharmaceutical revealed the positive results from its ongoing global Phase III GENEr8-1 study of Roctavian (valoctocogene roxaparvovec).   This is the largest and longest global Phase III study […]

The US Food and Drug Administration (FDA) has granted Orphan Drug Designation for iOnctura’s IOA-244, which is in development for solid and hematologic malignancies including uveal melanoma.  Uveal melanoma is a disease in which cancer originates in the tissues of the eye, causing symptoms such as blurred vision or a dark spot on the iris.   […]

Minoryx and Neuraxpharm have agreed a strategic alliance to provide a new therapy for rare CNS disease patients in Europe.  The companies have entered into a license agreement for the European rights to leriglitazone, currently under European Medicines Agency (EMA) review for the orphan indication X-linked Adrenoleukodystrophy (X-ALD).  Minoryx has granted Neuraxpharm exclusive European rights […]

Terebellum and NeurosolvTherapeutics will partner to launch Perineline, a new patented two-part therapy for spinal cord injuries.  The drug has attained EMA Orphan Designation and it is hoped that it will revolutionise the therapeutic treatment and resulting end patient outcomes from spinal cord injuries.   Terebellum will contribute strategic guidance in the pre-commercialisation stage, market access […]

The European Commission (EC) has granted marketing authorisation for Alnylam Pharmaceuticals’ AMVUTTRA (vutrisiran) for the treatment of hereditary transthyretin-mediated (hATTR) amyloidosis. The RNAi therapeutic has been approved in adult patients with stage 1 or stage 2 polyneuropathy, following positive 18-month results from the HELIOS-A Phase III study. AMVUTTRA significantly improved the signs and symptoms of […]