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FDA grants Fast Track Designation to rare disease treatment
The US Food and Drug Administration (FDA) has granted Fast Track Designation to a treatment for the rare paediatric disease late-infantile neuronal ceroid lipofuscinosis (LINCL/CLN2 disease).
PLX-200, developed by biotechnology company Polaryx Therapeutics, will now undergo an expedited review with the potential to address the paediatric disease with unmet need.
The treatment is an orally available compound comprised of gemfibrozil, an FDA-approved lipid regulating agent in the fibrate family. Preclinical trials show gemfibrozil can cross the blood-brain barrier, being considered safe for use in adults over several decades of clinical investigation.
“Receiving Fast Track Designation represents an important regulatory milestone as we prepare to initiate the SOTERIA Phase II basket trial evaluating PLX200 across multiple lysosomal storage disorders” said Alex Yang, Chair and Chief Executive Officer of Polaryx Therapeutics.
“We appreciate the FDA’s recognition of the potential of PLX200 to address the significant unmet medical need in CLN2 disease and look forward to continued engagement with the agency as we advance development of this programme.”
The post FDA grants Fast Track Designation to rare disease treatment appeared first on Drug Discovery World (DDW).
Can any of the new obesity medications in development stand out from the pack? Which company just broke records with its IPO? And will the Food and Drug Administration allow greater access to experimental peptides?
We discuss all that and more on this week’s episode of “The Readout LOUD,” STAT’s biotech podcast.
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RFK Jr. claims his calendar is publicly available. We’ve been trying to get it for a year
WASHINGTON — Health secretary Robert F. Kennedy Jr. on Wednesday pointed to his “publicly available calendar” as an example of his commitment to transparency and to beat back unfavorable reporting.
But no such calendar, detailing who Kennedy meets with or how he spends his time, has been released by the administration. STAT has been asking the Department of Health and Human Services for Kennedy’s calendar for more than a year, via Freedom of Information Act requests and emails to the press office.
WASHINGTON — Health secretary Robert F. Kennedy Jr. on Wednesday pointed to his “publicly available calendar” as an example of his commitment to transparency and to beat back unfavorable reporting.
But no such calendar, detailing who Kennedy meets with or how he spends his time, has been released by the administration. STAT has been asking the Department of Health and Human Services for Kennedy’s calendar for more than a year, via Freedom of Information Act requests and emails to the press office.
Uncategorized
Nonprofit buys experimental cancer drug to maintain patient access
In a rare move, nonprofit organization Blood Cancer United announced Thursday it was buying the remaining supplies of Luvelta, a discontinued investigational cancer drug.
As part of the transaction, Blood Cancer United, previously known as the Leukemia & Lymphoma Society, will also acquire the investigational new drug designation and manage the compassionate-use program for children with a rare form of blood cancer, distributing the medication to patients at no cost while supplies last.
In a rare move, nonprofit organization Blood Cancer United announced Thursday it was buying the remaining supplies of Luvelta, a discontinued investigational cancer drug.
As part of the transaction, Blood Cancer United, previously known as the Leukemia & Lymphoma Society, will also acquire the investigational new drug designation and manage the compassionate-use program for children with a rare form of blood cancer, distributing the medication to patients at no cost while supplies last.
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