Biologics

Orchard’s Leukodystrophy Gene Therapy OK’d

FDA has approved an Orchard Therapeutics BLA for Lenmeldy (atidarsagene autotemcel), a one-time, single-dose gene therapy indicated for treating children with pre-symptomatic late infantile, pre-symptomatic early juvenile or early symptomatic early juvenile metachromatic leukodystrophy (MLD), a rare genetic disease affecting the brain and nervous system. “It is caused by a deficiency of an enzyme called arylsulfatase A (ARSA), leading to a buildup of sulfatides (fatty substances) in the cells,” an FDA release says. “This buildup causes damage to the central and peripheral nervous system, manifesting with loss of motor and cognitive function and early death.”

Lenmeldy is made from a patient’s own hematopoietic stem cells that are genetically modified to include functional copies of the ARSA gene, according to the agency. “The modified stem cells are transplanted back into the patient where they engraft (attach and multiply) within the bone marrow,” it says. “The modified stem cells supply the body with myeloid (immune) cells that produce the ARSA enzyme, which helps break down the harmful build-up of sulfatides and may stop the progression of MLD.”

After cell therapy, FDA says patients should be monitored for neutrophil counts and risk of delayed platelet engraftment until engraftment has been achieved. “Treatment with Lenmeldy may be associated with formation of blood clots or a type of swelling of brain tissues known as encephalitis,” it says. “There is a potential risk of blood cancer associated with this treatment; however, no cases have been seen in patients treated with Lenmeldy. Patients receiving this product should have lifelong monitoring for hematologic malignancies, including a complete blood count (with differential) annually and integration site analysis, as warranted, for at least 15 years after treatment.”

Gupta Raises Concerns About Overton’s Independence, Ideology

FDA commissioner nominee Heidi Overton’s responses on vaccines, contraception and abortion medication during her 9/24 Senate confirmation hearing raise concerns that ideology could influence her decision-making if she takes over the agency, MSNBC senior medical analyst Dr. Vin Gupta said Friday. Gupta was particularly critical of Overton’

Overton Confirmation Remains Uncertain After Senate Hearing

FDA commissioner nominee Heidi Overton emerged from her Senate confirmation hearing Thursday (see earlier story) without resolving questions about whether she has enough Republican support to advance — and with a rapidly narrowing window for confirmation before the midterm elections. The Senate Health, Education, Labor and Pensions Committee hearing provided few

FDA Classifies Boston Scientific Imager II Catheter Recall as Class I

FDA has classified Boston Scientific's recall of its Imager II Angiographic Catheters as Class I because a manufacturing problem could cause catheter tips to degrade and detach, potentially resulting in life-threatening complications. The agency announced the Class I classification 9/25, updating an Early Alert it issued

FDA Approves Gazyva for Childhood-Onset Idiopathic Nephrotic Syndrome

FDA has approved Roche’s Gazyva (obinutuzumab) for reducing the risk of relapse in adults and children aged 2 and older with frequently relapsing or steroid-dependent, childhood-onset idiopathic nephrotic syndrome who are in remission. The expanded approval covers the use of the CD20-directed antibody beyond its existing

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Gupta Raises Concerns About Overton’s Independence, Ideology

Overton Confirmation Remains Uncertain After Senate Hearing

FDA Classifies Boston Scientific Imager II Catheter Recall as Class I

FDA Approves Gazyva for Childhood-Onset Idiopathic Nephrotic Syndrome