After almost 30 years in business, Sangamo Therapeutics Inc. is finally nearing a BLA filing for one of its programs. But the company, wounded by the recent loss of alliances with Biogen Inc. and Novartis AG, is also running out of cash and investor interest – and it badly needs a new deal to stay afloat.
Positive results from Bridgebio Pharma Inc.’s phase II study of infigratinib in children with achondroplasia, a genetic disease that inhibits bone length and leads to short stature, prompted the company stock to surge. Participants receiving the highest dosage, which was the fifth cohort getting 0.25 mg/kg daily, saw a 3.03-centimeter increase, about 1.19 inch, in their height annually, which produced a “p” value of 0.0022.
An uptick in positive clinical data reported in recent months has helped nudge the BioWorld Biopharmaceutical Index (BBI) to its highest point this year. BBI is up by 15.58%, significantly higher than the 1.36% increase recorded at the end of August, just as statistically significant clinical results began to roll in from a number of biopharma companies.
After winning the backing of European regulators, Biomarin Pharmaceutical Inc. is returning to the U.S. FDA with its hemophilia A gene therapy, valoctocogene roxaparvovec, following a rejection in August 2020. It's armed with data it hopes will assuage concerns about long-term safety and benefits.
Wall Street apparently wants to see longer-term data from Bridgebio Pharma Inc. with oral infigratinib in children with achondroplasia (ACH) before deciding about the drug’s chances against the approved therapy Voxzogo (vosoritide) from Biomarin Pharmaceutical Inc.
The first gene therapy to treat severe hemophilia A was among the drugs recommended for European approval by regulators from the EMA’s CHMP at its monthly meeting. Manufactured by Biomarin Pharmaceutical Inc., Roctavian (valoctocogene roxaparvovec) was recommended for conditional marketing authorization in the EU for severe hemophilia A in adults who do not have factor VIII inhibitors and no antibodies to adeno-associated virus serotype 5.
The recent start of Neurogene Inc.’s first-in-human trial testing NG-101 in ceroid lipofuscinosis type 5 (CLN5) Batten disease (BD) drew investor attention to the space, where a handful of developers vie for new therapies against the challenging indication.
PERTH, Australia – A new parliamentary report, The New Frontier: Delivering better health for all Australians, is recommending significant reforms to the nation’s health care system to ensure Australians have faster access to new drugs and devices. The House of Representatives Standing Committee on Health, Aged Care and Sport examined the range of new drugs and emerging medical technologies that are in development and progressing through the regulatory system in Australia and in other countries.
PERTH, Australia – A new parliamentary report, The New Frontier: Delivering better health for all Australians, is recommending significant reforms to the nation’s health care system to ensure Australians have faster access to new drugs and devices. The House of Representatives Standing Committee on Health, Aged Care and Sport examined the range of new drugs and emerging medical technologies that are in development and progressing through the regulatory system in Australia and in other countries.
Having unveiled more data from the ongoing, global phase III Gener8-1 study with Roctavian (valoctocogene roxaparvovec, also known as valrox), Biomarin Pharmaceutical Inc. remains on track to file a regulatory submission with the FDA in the second quarter of this year for the gene therapy to treat adults with severe hemophilia A. The EMA is already reviewing a marketing authorization application.
