Chiesi Farmaceutici SpA scored U.S. FDA clearance of the enzyme replacement therapy (ERT) Lamzede (velmanase alfa-tycv) for non-central nervous system manifestations of alpha-mannosidosis (AM) in adult and pediatric patients. An ultra-rare, progressive lysosomal storage disorder, AM is caused by deficiency in the enzyme alpha-mannosidase. Lamzede is the first ERT to win approval in the indication, characterized by an inability to properly break down certain groups of complex sugars in the body’s cells. Read More
The A’s have it but the B’s don’t in Moderna Inc.’s pivotal phase III study of mRNA-1010, a seasonal flu vaccine for adults living in the southern hemisphere. Interim results showed the vaccine achieved superiority on seroconversion rates for influenzas A/H3N2 and A/H1N1, superiority on geometric mean titer ratios for influenza A/H3N2 and noninferiority on geometric mean titer ratios for influenza A/H1N1. However, noninferiority was not met for the endpoints against the influenza B/Victoria- and B/Yamagata-lineage strains. Read More
Denali Therapeutics Inc.’s extensive update on clinical programs in central nervous system diseases at the start of this year included plans for lead asset DNL-310 in mucopolysaccharidosis II (MPS II), also known as Hunter syndrome – a space where other notable players include such names as Regenxbio Inc. and Takeda Pharmaceutical Co. Ltd. Read More
The stage is set for a showdown between the pharma industry and national governments and public health experts over which policy the EU should grasp, as it bids to create a pan-European incentive scheme that will encourage innovation and get more antibiotics through to market. At issue is a proposal supported by the industry, under which companies getting approval for a new antibiotic would be given a voucher allowing them to extend market exclusivity of any different drug of their choice for one year. Read More
One of the most important transformations in the pharmaceutical industry over the next decade is the ability to modify genes or expression and go after any target, Cargene Biopharma Inc. CEO Kathy He told BioWorld, explaining that small molecules and large molecules can only go after 15% of the known targets. But the technology platform of short-interfering RNA (siRNA) opens a huge opportunity to make those inaccessible targets available, she said. Read More
Ascletis Pharma Inc. presented subgroup data from a phase II trial showing its subcutaneous PD-L1 antibody envafolimab (ASC-22) was able to functionally cure chronic hepatitis B, according to hepatitis B surface antigen decline following 24-week treatment. Read More
San Diego-based Travere Therapeutics Inc. gained U.S. FDA accelerated approval for its dual endothelin angiotensin receptor antagonist, Filspari (sparsentan), to reduce proteinuria in adults with primary IgA nephropathy, or Berger’s disease. Read More
New and updated clinical data presented by biopharma firms at the American Society of Clinical Oncology Genitourinary Cancers Symposium, including: Bellicum, Celcuity, Janssen, Lava, Promontory. Read More
Regulatory snapshots, including global drug submissions and approvals, clinical trial approvals and other regulatory decisions and designations: Axcella, Entera, Kancera, Nymox, Ose, Pharming, Redhill. Read More
