drug · small molecule

ALYFTREK

updated 5mo ago

by $VERX Vertex, Inc.🔗 ChEMBL

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Small molecule drug with a maximum clinical stage of Approval, with an approval for cystic fibrosis.

targets

Cystic fibrosis transmembrane conductance regulator

all catalysts(0)

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Trials studying ALYFTREK

phase1Evaluation of VX-828 in Healthy Participants and in Participants With Cystic Fibrosisrecruitingn=255

News(5)

ReCode CEO Suliman leaves cystic fibrosis mission in mentee’s hands
After achieving clinical validation earlier this year, ReCode Therapeutics has called up a new CEO to take on the mission of advancing programs for cystic fibrosis and primary ciliary dyskinesia th…
biospace · 10d ago
Lilly pens $370M deal with OmniAb for ion channel collab, but details remain vague
Antibody platform business OmniAb continues to rack up Big Pharma partnerships, with Eli Lilly signing off on up to $370 million biobucks tied to an ion channel program.
fiercebiotech · 1mo ago
Sionna’s cystic fibrosis failure is a windfall for Vertex’s blockbuster franchise
A surprise flop in Phase 2a cystic fibrosis trial sent Sionna’s shares plummeting, but for Vertex, the news reinforced its standard-of-care standing with highly successful drugs like Trikafta.
biospace · 1mo ago
Vertex’s biggest overhang: a rival 50 times smaller
Despite another strong earnings report, analysts warned that upcoming cystic fibrosis data from Sionna Therapeutics "looms large" over Vertex.
biopharma_dive · 2mo ago
Vertex earnings get muted investor response
While overall revenue fell just shy of Wall Street forecasts, sales of the gene editing medicine Casgevy and pain drug Journavx missed analyst estimates by double-digit percentages.
biopharma_dive · 5mo ago

brand

ALYFTREK

trade names

Deutivacaftor component of alyftrek

generic

DEUTIVACAFTOR

MOA

12.1 Mechanism of Action Vanzacaftor and tezacaftor bind to different sites on the CFTR protein and have an additive effect in facilitating the cellular processing and trafficking of select mutant forms of CFTR (including F508del- CFTR) to increase the amount of CFTR protein delivered to the cell surface compared to either molecule alone. Deutivacaftor potentiates the channel open probability (or gating) of the CFTR protein at the cell surface. The combined effect of vanzacaftor, tezacaftor and deutivacaftor is increased quantity and function of CFTR at the cell surface, resulting in increased CFTR activity as measured both by CFTR-mediated chloride transport in vitro and by sweat chloride in patients with CF. CFTR Chloride Transport Assay in Fischer Rat Thyroid Cells Expressing Mutant CFTR Protein Effects of vanzacaftor/tezacaftor/deutivacaftor on chloride transport for mutant CFTR protein was determined in Ussing chamber electrophysiology studies using a panel of Fischer Rat Thyroid (FRT) cell lines stably expressing CFTR protein from individual variants. Vanzacaftor/tezacaftor/deutivacaftor increased chloride transport in FRT cells expressing select CFTR variants, as identified in Table 5. The threshold that the treatment-induced increase in chloride transport must exceed for the mutant CFTR protein to be considered responsive is ≥10% of normal over baseline. This threshold was used because it is expected to predict clinical benefit. For individual variants, the magnitude of the net change over baseline in CFTR-mediated chloride transport in vitro is not correlated with the magnitude of clinical response. CFTR Chloride Transport Assay in Human Bronchial Epithelial Cells Expressing Mutant CFTR Protein Homozygous and heterozygous N1303K- Human Bronchial Epithelial (HBE) cells showed greater chloride transport in the presence of vanzacaftor/tezacaftor/deutivacaftor than F508del/F508del- HBE cells treated with tezacaftor/ivacaftor which has shown clinical benefit in

indication

1 INDICATIONS AND USAGE ALYFTREK is indicated for the treatment of cystic fibrosis (CF) in adult and pediatric patients 6 years of age and older who have a clinical diagnosis of CF and who have at least one variant in the cystic fibrosis transmembrane conductance regulator ( CFTR ) gene that is either responsive based on clinical and/or in vitro data (see Table 5 ) or results in production of CFTR protein [see Clinical Pharmacology (12.1) ]. If the patient's genotype is unknown, an FDA-cleared CF genetic test should be used to confirm the presence of at least one variant in the CFTR gene that is either responsive based on clinical and/or in vitro data or results in production of CFTR protein. ALYFTREK is a combination of deutivacaftor, a CFTR potentiator, tezacaftor, and vanzacaftor indicated for the treatment of cystic fibrosis (CF) in adult and pediatric patients aged 6 years and older who have a clinical diagnosis of CF and who have at least one variant in the CFTR gene that is either responsive based on clinical and/or in vitro data or results in production of CFTR protein. ( 1 , 12.1 ) If the patient's genotype is unknown, an FDA-cleared CF genetic test should be used to confirm the presence of at least one variant in the CFTR gene that is either responsive based on clinical and/or in vitro data or results in production of CFTR protein. ( 1 )

Data sources

ClinicalTrials.gov · OpenTargets

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News(5)

ReCode CEO Suliman leaves cystic fibrosis mission in mentee’s hands

After achieving clinical validation earlier this year, ReCode Therapeutics has called up a new CEO to take on the mission of advancing programs for cystic fibrosis and primary ciliary dyskinesia th…

biospace · 10d ago

Lilly pens $370M deal with OmniAb for ion channel collab, but details remain vague

Antibody platform business OmniAb continues to rack up Big Pharma partnerships, with Eli Lilly signing off on up to $370 million biobucks tied to an ion channel program.

fiercebiotech · 1mo ago

Sionna’s cystic fibrosis failure is a windfall for Vertex’s blockbuster franchise

A surprise flop in Phase 2a cystic fibrosis trial sent Sionna’s shares plummeting, but for Vertex, the news reinforced its standard-of-care standing with highly successful drugs like Trikafta.

biospace · 1mo ago

Vertex’s biggest overhang: a rival 50 times smaller

Despite another strong earnings report, analysts warned that upcoming cystic fibrosis data from Sionna Therapeutics "looms large" over Vertex.

biopharma_dive · 2mo ago

Vertex earnings get muted investor response

While overall revenue fell just shy of Wall Street forecasts, sales of the gene editing medicine Casgevy and pain drug Journavx missed analyst estimates by double-digit percentages.

biopharma_dive · 5mo ago

ALYFTREK

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targets

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Trials studying ALYFTREK

News(5)

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ALYFTREK

🐶 often sniffed alongside

targets

all catalysts(0)

Trials studying ALYFTREK

News(5)

Report a data issue

comments(0)