Alnylam gets NICE backing for porphyria therapy GivlaariUK cost-effectiveness watchdog NICE is set to recommend NHS use of Alnylam’s gene-silencing therapy Givlaari in England and Share XAlnylam gets NICE backing for porphyria therapy Givlaarihttps://pharmaphorum.com/news/alnylam-gets-nice-backing-for-porphyria-therapy-givlaari/
NICE changes stance on Sanofi’s Dupixent for severe asthma18 months after saying “no” to regular NHS funding for Sanofi and Regeneron’s Dupixent (dupilumab) for severe asthma Share XNICE changes stance on Sanofi’s Dupixent for severe asthmahttps://pharmaphorum.com/news/nice-changes-stance-on-sanofis-dupixent-for-severe-asthma/
Flash FDA approval sends ChemoCentryx spiralling upwardsChemoCentryx has claimed FDA approval for its avacopan drug for a rare autoimmune disease, becoming the first orally-active Share XFlash FDA approval sends ChemoCentryx spiralling upwardshttps://pharmaphorum.com/news/flash-fda-approval-sends-chemocentryx-spiralling-upwards/
Joy as sickle cell patients get access to Novartis’ Adakveo after NICE rulingPeople living with sickle cell disease (SCD) in England and Wales will be able to get access to Share XJoy as sickle cell patients get access to Novartis’ Adakveo after NICE rulinghttps://pharmaphorum.com/news/joy-as-sickle-cell-patients-get-access-to-novartis-adakveo-after-nice-ruling/
AZ claims full control of Caelum’s AL amyloidosis drug in $500m dealAstraZeneca’s newly acquired Alexion will buy the remaining equity in rare disease specialist Caelum Biosciences in a deal Share XAZ claims full control of Caelum’s AL amyloidosis drug in $500m dealhttps://pharmaphorum.com/news/az-claims-full-control-of-caelums-al-amyloidosis-drug-in-500m-deal/
Bad news for Biohaven as verdiperstat flunks first phase 3 testBiohaven’s key pipeline drug verdiperstat has failed to have any Therapeutic effect in patients with the rare neurological Share XBad news for Biohaven as verdiperstat flunks first phase 3 testhttps://pharmaphorum.com/news/bad-news-for-biohaven-as-verdiperstat-flunks-first-phase-3-test/
NICE backs BioCryst’s Orladeyo for hereditary angioedemaBioCryst Pharma’s Orladeyo has been recommended by NICE as a treatment for the rare disease hereditary angioedema (HAE) Share XNICE backs BioCryst’s Orladeyo for hereditary angioedemahttps://pharmaphorum.com/news/nice-backs-biocrysts-orladeyo-for-hereditary-angioedema/
Astellas reports fourth death in halted gene therapy trialThere has been a fourth patient fatality in Astellas’ clinical trial of its AT132 gene therapy for the Share XAstellas reports fourth death in halted gene therapy trialhttps://pharmaphorum.com/news/astellas-reports-fourth-death-in-halted-gene-therapy-trial/
FDA slaps clinical hold on BioMarin’s PKU gene therapyBioMarin Pharma has had another setback in its gene therapy development programme, announcing this morning that the FDA Share XFDA slaps clinical hold on BioMarin’s PKU gene therapyhttps://pharmaphorum.com/news/fda-slaps-clinical-hold-on-biomarins-pku-gene-therapy/
