FDA Approves Gene Therapy For Rare Disorder After Rejecting It 2 Years Ago
The therapy marketed as Kresladi was initially rejected in 2024 because of manufacturing concerns. It treats leukocyte adhesion deficiency type 1 (LAD-1), an ultra-rare disease — affecting about one in a million — that makes children vulnerable to life-threatening infections and can lead to death.
Stat:
Rocket Gene Therapy Kresladi Approved For Rare Immune Disorder
The Food and Drug Administration approved on Thursday a gene therapy for severe leukocyte adhesion deficiency type 1 (LAD-1), an ultra-rare disease that leaves children vulnerable to life-threatening infections. (Mast, 3/26)
More pharmaceutical industry news —
Healthcare Dive:
CVS, FTC Reach Proposed Settlement In Insulin Pricing Case
CVS Health has reached a proposed settlement with the Federal Trade Commission in the agency’s sweeping case against major pharmacy benefit managers for allegedly inflating the cost of U.S. insulin. The proposed consent agreement was disclosed Monday in a joint motion from the FTC and CVS for the company’s subsidiaries, PBM Caremark and group purchasing organization Zinc, to withdraw from the case while antitrust regulators consider the deal. (Parduhn, 3/24)
Bloomberg:
Novartis To Buy Excellergy In Deal Worth Up To $2 Billion
Novartis AG has agreed to acquire biotech firm Excellergy Inc. for as much as $2 billion, in a deal that will strengthen its portfolio of treatments for food allergies and other immunology conditions. The transaction, which includes upfront and milestone payments, is expected to close in the second half of this year, subject to conditions including regulatory approvals, the Swiss drugmaker said in a statement. (3/27)
Bloomberg:
Nucleus Genomics Expands Embryo Testing To India, Middle East Clinics
A New York-based embryo testing startup has struck deals with IVF clinics in India and the Middle East to expand use of the controversial technology. Nucleus Genomics Inc. has agreements with Indira IVF, which runs more than 165 fertility clinics across India, and Abu Ghosh Fertility Group, which operates in Saudi Arabia and Jordan, the company said Thursday. As part of the deals, the clinics will offer Nucleus’ tests to couples undergoing in vitro fertilization. Nucleus also has an existing partnership with an operator of IVF clinics in Mexico. (Smith, 3/26)
MedPage Today:
Can GLP-1 Drugs Prevent Worsening Mental Illness? Perhaps, Study Suggests
People with pre-existing depression or anxiety were less likely to have mental health worsening while taking a GLP-1 medication, observational Swedish data suggested. (Monaco, 3/26)
MedPage Today:
Are Bayesian Statistics Coming To A Clinical Trial Near You?
The FDA's move to incorporate Bayesian statistical methods into clinical trials of drugs and biologics garnered special treatment in JAMA this week. JAMA published three perspectives -- two welcoming the agency's draft guidance, published in January, and one urging caution. (Fiore, 3/26)
The latest on Elizabeth Holmes —
Bloomberg:
Elizabeth Holmes Gets A Year Knocked Off Her Prison Sentence
Disgraced Theranos Inc. founder Elizabeth Holmes got a year knocked off her 11 1/4-year prison term under a change in sentencing guidelines that applies to convicts with no previous criminal history. Holmes qualified for the reduced term under a 2023 rule change allowing first-time offenders to do less time for some non-violent crimes, according to an order issued Thursday by the federal judge who sentenced her in 2022 for defrauding investors in her blood-testing startup. (Hurtado and Blumberg, 3/26)
