Winst gevend schreef op 6 augustus 2023 11:47:
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Assessment of drug approval process in this class of drugs might also lead
to more stringent procedures concerning the development of
other cancer drugs.
As the path forward might require randomized control trial as a standard for initial approval,
these trials would also necessitate the inclusion of existing relevant standard of care therapies
in their placebo/non-intervention arm.
However, withdrawal of several approvals should not mean a death sentence for all PI3Ki,
as we have learned from the long and windy road for approval of gemtuzumab ozogamicin
in
acute myelogenous leukemia and gefitinib in
EGFR mutated non-small cell lung cancer.In this regard, while acknowledging the low number of patients,
recent data from the novel PI3Kd inhibitor, leniolisib are showing encouraging safety profile
up to 5 years in patients with activated PI3Kd syndrome (APDS)
with only one discontinuation thought not to be drug-related.
Kortom, de 2e indicatie van Leniolisib blijft voorlopig een groot geheim
waarvan drie partijen op de hoogte zijn, Pharming, Novartis en de FDA.