These drugs target enzymes that are
involved in the regulation of gene expression.
The European Medicines Agency has given its recommendation for Bayer’s and
Lilly’s Vitrakvi for the treatment of NTRK fusion-positive cancers.
Vitrakvi (larotrectinib) has received FDA’s nod last year in the month of November. It is a drug which targets the tumour based on genetic markers for cancers that are a result of a genomic abnormality called NTRK fusion.
Originally co-developed by Bayer
and Loxo Oncology, the latter which was afterwards acquired by Lilly for an
amount of USD 8Billion.
Vitrakvi is a second drug after Merck’s Keytruda that is tumour-agnostic. Roche’s Rozlytrek (entrectinib) became the third drug to get approval from the FDA to target NTRK fusion and ROS1 in lung cancer.
Bluebird Bio, a biotechnology firm has reported positive results from
the Phase II/III clinical trials of Lenti-D gene therapy to target cerebral
adrenoleukodystrophy (CALD).
CALD, a rare genetic disorder can
result in severe loss of neurologic function and death.
The Starbeam trial, intended to gauge
the safety and efficacy of Lenti-D in boys aged 17 and under. Its primary
efficacy endpoint is the proportion of patients alive and free of major
functional disabilities (MFDs) at 24 months.
The results were primarily based
on the proportion of patients alive and free of major functional disabilities
(MFDs) at 24 months.
The data revealed that 88% of
patients who underwent therapy and stayed under supervision for 24 months were
free of major functional disabilities (MFDs) at two years and remained the same
for up to five years.
MFDs are a group of symptoms comprising loss of communication ability, total incontinence, and complete loss of voluntary movement, the need for tube feeding, cortical blindness and wheelchair dependence. The company is also running observational study of allogeneic hematopoietic stem cell transplant (allo-HSCT) in CALD patients which gave favourable results.
