NEW YORK – Krystal Biotech this week said it has sold a rare pediatric disease priority review voucher to an undisclosed party for $100 million. The US Food and Drug Administration awarded the Pittsburgh-based firm a priority review voucher after granting accelerated approval to Vyjuvek (beremagene geperpavec-svdt), a gene therapy for patients at least 6 months old with recessive or dominant dystrophic epidermolysis bullosa caused by COL7A1 gene mutations. The FDA gives these vouchers to encourage development of new rare disease treatments, and companies can use them for a different product application, sell them, or transfer them.
The federal Defense Health Agency announced this week a five-year extension of the Laboratory Developed Tests demonstration from July 19, 2023, through July 18, 2028. Anyone currently covered by the military health system TRICARE will continue to have access to certain LDTs. Additionally, the DHA said that TRICARE will now cover several preconception and prenatal carrier screening tests, which previously had been offered through the demonstration, as part of the basic TRICARE benefit. LDTs now covered by TRICARE include tests for cystic fibrosis, spinal muscular atrophy, fragile X syndrome, Tay-Sachs disease, hemoglobinopathies, and conditions linked with Ashkenazi Jewish descent. TRICARE is retroactively covering the tests as screenings back to Dec. 27, 2021, and will cover one test per condition per lifetime, the DHA said.
The UK National Institute for Health and Care Excellence (NICE) this week recommended the use of fecal immunochemical tests to guide referrals of patients suspected of having colorectal cancer. The recommendation, which covers a variety of patients, follows similar recommendations from its diagnostic advisory committee in July, and is being made to reduce the number of unnecessary colonoscopies, NICE said.
Verismo Therapeutics this week said that it has licensed two anti-CD19 binders developed under a 2022 research agreement between Verismo and the University of Pennsylvania. Verismo is using one of the binders, DS191, to develop a CAR T-cell immunotherapy for blood cancer, for which it plans to submit an investigational new drug application by the end of 2023. Both binders are available for future partnering.
Amgen said this week that the US Food and Drug Administration will hold an advisory committee meeting to review data supporting the full approval of Lumakras (sotorasib) in previously treated locally advanced or metastatic KRAS G12C-mutated non-small cell lung cancer patients. The FDA granted accelerated approval for Lumakras in this setting in May 2021. The advisory committee meeting will be on Oct. 5, and the FDA will decide whether to grant full approval to Lumakras by Dec. 24.
Bloomsbury Genetic Therapies this week said the US Food and Drug Administration has granted its investigational gene therapy for Niemann-Pick disease type C rare pediatric disease designation. NPC, an inherited neurodegenerative disease largely due to NPC1 gene mutations, impairs patients' speech and mobility and causes epilepsy and dementia. London-based Bloomsbury is developing BGT-NPC, an adeno-associated virus 9-based gene therapy designed to promote enhanced NPC1 protein expression, as a potential cure for the rare disease. The company is wrapping up preclinical studies on the drug. In June, the UK Medicines and Healthcare Products Regulatory Agency approved Bloomsbury's plans to conduct a toxicology and biodistribution study in rodents before commencing a Phase I/II/III clinical trial.
The Japanese Ministry of Health, Labour, and Welfare approved an expanded indication for AstraZeneca's Soliris (eculizumab) to include pediatric patients with generalized myasthenia gravis (gMG) who are anti-acetylcholine receptor antibody-positive and whose symptoms are difficult to control with high-dose intravenous immunoglobulin therapy or plasmapheresis, the company said this week. Soliris, a C5 complement inhibitor, was initially approved by the Japanese agency in 2017 as a treatment for certain adults with gMG, a rare chronic autoimmune neuromuscular disease.
XNK Therapeutics this week began a research collaboration with Karolinska University Hospital in Sweden to evaluate its autologous natural killer cell therapy candidate XNK02 in acute myeloid leukemia (AML). The partners will conduct a large-scale bioreactor culture study of AML patients' immune cells to evaluate the expansion procedure, the properties of the expanded natural killer cells, and the cells' ability to kill tumor cells ex vivo.
The US Food and Drug Administration this week granted orphan drug designation to Nexcella's autologous CAR T-cell therapy, NXC-201, for the treatment of multiple myeloma. Nexcella is studying NXC-201 as an outpatient treatment for multiple myeloma in a Phase I trial. The FDA grants orphan designation to drugs for rare diseases, which provides sponsors tax credits for clinical trials, exemption from user fees, and seven years of market exclusivity for the drug after regulatory approval.
Taysha Gene Therapies this week said the US Food and Drug Administration has granted fast-track status to TSHA-102, an adeno-associated vector 9 gene therapy it is studying in Rett syndrome. TSHA-102 is designed to mediate MECP2 levels in the central nervous system on a cell-by-cell basis and limit the risk of overexpression. With fast-track status, Taysha can pursue expedited regulatory review for TSHA-102 and interact frequently with the FDA about its regulatory application for the agent. The FDA and the European Commission consider TSHA-102 an orphan drug, and the FDA has also given it a rare pediatric disease designation. Earlier this month, an independent data monitoring committee told the Dallas-based firm it could dose a second patient in the Phase I/II REVEAL trial, in which researchers are studying TSHA-102's safety and efficacy in adult women with Rett syndrome caused by loss-of-function mutations in the MECP2 gene.
In Brief This Week is a selection of news items that may be of interest to our readers but had not previously appeared in Precision Medicine Online.