Vertex CEO hints at ‘new ideas’ to bring Orkambi to UK patientsVertex CEO Jeffrey Leiden has offered an olive branch to negotiators in the UK that could see the Share XVertex CEO hints at ‘new ideas’ to bring Orkambi to UK patientshttps://pharmaphorum.com/news/vertex-ceo-hints-at-new-ideas-to-bring-orkambi-to-uk-patients/
Akcea/Ionis’ rare disease drug Waylivra set for EU market after rejection in USEuropean regulators have recommended a licence for Akcea and Ionis’ ultra-rare disease drug Waylivra, a therapy that the Share XAkcea/Ionis’ rare disease drug Waylivra set for EU market after rejection in UShttps://pharmaphorum.com/news/akcea-ionis-rare-disease-drug-waylivra-set-for-eu-market-after-rejection-in-us/
Deep Dive: R&D InnovationIn this issue of Deep Dive we look at innovation in pharma R&D, asking how can pharmaceutical companies Share XDeep Dive: R&D Innovationhttps://pharmaphorum.com/r-d/pharma-research-rare-diseases-0219/
Rare Disease Day: How the patient voice can tear down barriersTo have any impact, patient engagement needs to start with the drug development pathway, not at the point Share XRare Disease Day: How the patient voice can tear down barriershttps://pharmaphorum.com/views-and-analysis/rare-disease-day-how-the-patient-voice-can-tear-down-barriers/
Alexion aims for fourth rare disease use for blockbuster SolirisThe FDA has granted a fast review for Alexion Pharmaceuticals’ filing for its Soliris, as a treatment for Share XAlexion aims for fourth rare disease use for blockbuster Solirishttps://pharmaphorum.com/news/alexion-aims-for-fourth-rare-disease-use-for-blockbuster-soliris/
Ipsen to buy rare disease firm Clementia for up to $1.31bnFrance’s Ipsen has agreed to buy Canadian rare diseases biotech Clementia Pharmaceuticals in a deal worth up to Share XIpsen to buy rare disease firm Clementia for up to $1.31bnhttps://pharmaphorum.com/news/ipsen-to-buy-rare-disease-firm-clementia-for-up-to-1-31bn/
Roche to buy gene therapy firm Spark for $4.8 billionRoche is to buy gene therapy firm Spark Therapeutics for $4.8 billion, adding an already-approved treatment for inherited Share XRoche to buy gene therapy firm Spark for $4.8 billionhttps://pharmaphorum.com/news/roche-to-buy-spark-therapeutics/
Catalyst defends huge US price hike for rare disease drugCatalyst Pharmaceuticals has defended hiking the price of the rare disease drug Firdapse (amifampridine) from virtually nothing to Share XCatalyst defends huge US price hike for rare disease drughttps://pharmaphorum.com/news/catalyst-defends-huge-us-price-hike-for-rare-disease-drug/
Cell therapy produces encouraging first results in eye trialUK-based biotech ReNeuron has announced encouraging results from an early stage trial of its cell therapy for the Share XCell therapy produces encouraging first results in eye trialhttps://pharmaphorum.com/news/reneuron-cell-therapy-produces-encouraging-results-in-early-eye-trial/
