BridgeBio Pharma, a US-based biopharmaceutical company focused on genetic diseases, has received the US Food and Drug Administration (FDA) approval for Attruby (acoramidis).
Attruby is an orally administered near-complete stabiliser of Transthyretin (TTR), indicated to reduce cardiovascular death and cardiovascular-related hospitalisation.
The drug works by imitating a naturally occurring ‘rescue mutation’ of the TTR gene that targets the root cause of ATTR-CM, the destabilisation of the native TTR tetramer.
According to BridgeBio, Attruby is the only near-complete stabiliser of TTR approved in the US to treat adult patients with ATTR-CM to reduce cardiovascular death and hospitalisation.
BridgeBio Pharma founder and CEO Neil Kumar said: “With the landmark approval of Attruby, we gain the ability to serve patients with ATTR-CM.
“I’m grateful to each trial participant, their families, and the physicians, scientists and our team at BridgeBio who made this possible.
“Our journey is not over as we look to pursue approvals globally, next in Europe, Japan, and Brazil, and to continue exploring the full potential of this treatment. I am thrilled to extend our mission of ‘putting patients first’ with this third FDA approval in less than 10 years.”
The FDA approval is based on positive results from the Phase 3 ATTRibute-CM study, in which Attruby reduced cardiovascular death and hospitalisation, and improved quality of life.
The Phase 3 study enrolled 632 participants with symptomatic ATTR-CM, associated with either wild-type or variant TTR.
The study met its primary endpoint, a four-component composite endpoint, and showed a statistically significant treatment effect at 30 months.
The primary endpoint includes ACM, CVH, and N-terminal prohormone of brain natriuretic peptide (NT-proBNP), and a six-minute walk distance with a Win Ratio of 1.8.
In the study, the drug was generally well-tolerated, with the most common side effects being mild, including diarrhoea and abdominal pain.
In addition, BridgeBio is offering a patient support services programme, dubbed ForgingBridges, to help US patients and their families in accessing treatment.
Mayo Clinic MD Martha Grogan said: “Transthyretin cardiac amyloidosis is a progressive disease with a poor prognosis when left untreated.
“Having a new first-line treatment option which provides excellent TTR stabilisation and improves outcomes in this disease gives patients more options.
“Encouraging data suggests Attruby reduces all-cause mortality and cardiovascular hospitalisation as early as three months after initiation of therapy.
“With continued advances in therapy, this previously fatal disease is becoming a manageable chronic cardiovascular condition.”
