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.
Regulatory snapshots, including global drug submissions and approvals, clinical trial approvals and other regulatory decisions and designations: Axcella, Entera, Kancera, Nymox, Ose, Pharming, Redhill.
The European Parliament (EP) has voted to affirm the proposal by the European Commission (EC) to extend the compliance timelines for the Medical Device Regulation (MDR) in an urgent vote that eliminated any chance of debate. The vote was a landslide, coming in at 564 votes in support and three nays.
The U.K. government recently released its first ever medical technology strategy which aims to accelerate access to innovative medical technologies in the NHS. Although it was welcomed by the industry, and questions remain as to whether it goes far enough to address some of the challenges to the adoption of medical devices, eyes are on its implementation and whether it will achieve its aims.
If the Feb. 16 hearing before the U.S. Senate Commerce Committee is anything to go by, it’s almost a given that the bipartisan Pharmacy Benefit Manager (PBM) Transparency Act will eventually be passed by the Senate. But its journey through the Republican-controlled House could be more uncertain in light of growing concerns about an “activist agenda” at the FTC.
The U.S. FDA granted Nanjing Iaso Biotherapeutics Co. Ltd. both regenerative medicine advanced therapy and fast track designations for its new drug, BCMA CAR T-cell therapy CT-103A (equecabtagene autoleucel), allowing it to speed up development and commercialization in the U.S. for the treatment of relapsed/refractory multiple myeloma.
Regulatory snapshots, including global submissions and approvals, clinical trial approvals and other regulatory decisions and designations: Lumendi, Perfuze, Saniva, Zephyrus.
Regulatory snapshots, including global drug submissions and approvals, clinical trial approvals and other regulatory decisions and designations: Accord, Concert, Galera, Henlius, Kinnate, Redhill, Unravel.
The False Claims Act is perhaps the primary vehicle for U.S. federal authorities to extract penalties and fines from life science companies for violations of the law, but a new report by Gibson Dunn & Crutcher LLP suggests a mixed signal. The report notes that the U.S. Department of Justice (DOJ) recovered $2.2 billion via the FCA last year, but while that is not a conspicuous number for the past decade plus, what was conspicuous was that those sums were recovered by the second highest level of FCA actions settlement in the history of the FCA, suggesting that DOJ is keen on enforcement with no regard to the size of the target.
The Feb. 13-14 meeting of the U.S. Medicare Evidence Development and Coverage Advisory Committee (MedCAC) was the second step in an effort by CMS to overhaul the coverage with evidence development (CED) mechanism. While the meeting took place with the overhang of the issue of coverage for U.S. FDA-designated breakthrough devices, it seems there are changes coming to the CED program regardless of the breakthrough devices question, such as the imposition of CED study milestones that would presumably thwart the never-ending CED study per a vote held during the hearing.
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