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Vertex Pharmaceuticals

All articles tagged with #vertex pharmaceuticals

Gene Therapies for Sickle Cell Disease Face Access Barriers Despite High Efficacy
health4 days ago

Gene Therapies for Sickle Cell Disease Face Access Barriers Despite High Efficacy

The FDA approved Casgevy and Lyfgenia in 2023 as the first gene therapies for severe sickle cell disease, offering potential cures with 88% to 94% efficacy. However, uptake remains low, with only 64 patients receiving Casgevy and 115 receiving Lyfgenia by late 2025. Barriers include high costs ranging from $2.2 million to $3.1 million, complex treatment processes causing infertility, and limited provider availability. While the CMS Cell and Gene Therapy Access Model aims to lower costs, uneven coverage and geographic gaps persist, leaving many eligible patients without access.

via CNBC|
#gene-therapy#health#healthcare-access
Cystic Fibrosis’s Final 10% Face Treatment Gap as New Therapies Stall
health-and-science11 days ago

Cystic Fibrosis’s Final 10% Face Treatment Gap as New Therapies Stall

While 90% of cystic fibrosis (CF) patients benefit from Vertex Pharmaceuticals’ drugs Trikafta and Alyftrek, approximately 10% lack the specific genetic mutations required for these treatments to work. Emily’s Entourage, a nonprofit founded by patient Emily Kramer-Golinkoff, is funding research into alternative therapies, including gene treatments and antimicrobials, to address this gap. Recent setbacks, including halted trials by Vertex/Moderna and Boehringer Ingelheim, have delayed progress, but new approaches like Spirovant Sciences’ gene therapy and RNA-based methods offer hope. The foundation has raised over $22 million to support 51 projects, aiming to prevent the misconception that CF is fully cured and to accelerate treatments for rare genetic conditions.

via CNBC|
#clinical-trials#cystic-fibrosis#gene-therapy
Vertex Sues US for Fertility Support in Casgevy Gene Therapy
health2 years ago

Vertex Sues US for Fertility Support in Casgevy Gene Therapy

Vertex Pharmaceuticals has filed a lawsuit against the U.S. government to allow the company to fund fertility preservation services for patients receiving Casgevy, a CRISPR-based treatment for sickle cell disease. The lawsuit challenges an oral opinion from the Health and Human Services Office of the Inspector General, which deemed the program a violation of federal anti-kickback laws. Vertex argues that the program is essential for patients who must undergo chemotherapy, which can cause infertility, and that the refusal to allow such support exacerbates healthcare inequalities.

via STAT|
#anti-kickback-laws#crispr#fertility-preservation
"Vertex Pharmaceuticals Acquires Kidney Disease Drug Developer for $4.9 Billion"
business2 years ago

"Vertex Pharmaceuticals Acquires Kidney Disease Drug Developer for $4.9 Billion"

Vertex Pharmaceuticals announced its acquisition of Alpine Immune Sciences for $4.9 billion, causing Alpine's stock to surge by 37%. The deal is expected to close later this quarter and values Alpine stock at $65 per share, representing a 67% premium to Tuesday's close. Analysts believe Alpine's potential best-in-class treatment for IgA nephropathy, povetacicept, has the potential to lead its class due to its superior APRIL/BAFF inhibition and convenient dosing regimen. The acquisition reflects Vertex's confidence in Alpine's drug and its potential in treating autoimmune conditions. Vertex's stock also rose 1% following the announcement.

via Investor's Business Daily|
#acquisition#alpine-immune-sciences#business
"Inflation Report Sparks Stock Market Turbulence: Fed's Data Dependence Questioned"
finance2 years ago

"Inflation Report Sparks Stock Market Turbulence: Fed's Data Dependence Questioned"

Stock futures dipped as traders anticipated another inflation report, with Dow Jones Industrial Average, S&P 500, and Nasdaq-100 futures all sliding. The market reacted to a hot March inflation reading, prompting concerns about potential Federal Reserve rate cuts. The next key inflation tests come with March's producer price index and weekly jobless claims. Additionally, Vertex Pharmaceuticals announced its acquisition of Alpine Immune Sciences, causing shares to fluctuate.

via CNBC|
#earnings-season#federal-reserve#finance
"Vertex Pharmaceuticals' $4.9 Billion Acquisition of Alpine Immune Sciences Targets Kidney Disease Treatment"
business2 years ago

"Vertex Pharmaceuticals' $4.9 Billion Acquisition of Alpine Immune Sciences Targets Kidney Disease Treatment"

Vertex Pharmaceuticals Inc. is set to acquire Alpine Immune Sciences Inc. for approximately $4.9 billion, marking its largest acquisition to date. The deal will see Vertex pay $65 per share in cash for Alpine, a 67% premium over its closing price on Tuesday. The acquisition comes as Vertex looks to expand its portfolio with Alpine's immune treatments, while Alpine's stock surged 21% following reports of the potential sale.

via Bloomberg|
#acquisition#alpine-immune-sciences#biotech
"Vertex Pharmaceuticals' $4.9 Billion Bet on Kidney Disease Treatment with Alpine Immune Acquisition"
business-healthcare2 years ago

"Vertex Pharmaceuticals' $4.9 Billion Bet on Kidney Disease Treatment with Alpine Immune Acquisition"

Vertex Pharmaceuticals is acquiring Alpine Immune Sciences for $4.9 billion in cash, gaining access to the biotech firm's treatment for IgA nephropathy, an autoimmune disease of the kidney. The deal will provide Vertex with access to Alpine's povetacicept, a mid-stage treatment for IgAN, which targets proteins contributing to autoimmune and antibody diseases. The acquisition aligns with Vertex's focus on expanding its portfolio beyond cystic fibrosis and blood disorders, and the deal is expected to close in the second quarter of 2024.

via CNBC|
#acquisition#alpine-immune-sciences#autoimmune-disease
"South Africans Fight for Access to Life-Saving Cystic Fibrosis Drug"
health2 years ago

"South Africans Fight for Access to Life-Saving Cystic Fibrosis Drug"

South African patients with cystic fibrosis are taking legal action against Vertex Pharmaceuticals for access to the life-changing drug Trikafta, which costs $326,000 a year per patient. The lawsuit accuses the company of patent abuse and violating patients' human rights, as the drug is unaffordable for most. While Vertex defends its intellectual property rights, patients are resorting to importing a generic version from Argentina at a fraction of the cost. The legal battle highlights the challenges of accessing expensive medication in low- and middle-income countries, leaving many patients without life-saving treatment.

via The Guardian|
#cystic-fibrosis#health#patent-abuse