In 1922, a 14-year-old boy in Toronto, Canada, received the first injection of insulin to treat life-threatening diabetes. The same year, researchers began to test oral insulin formulations, hoping to relieve people with diabetes of daily shots. That effort failed, as have dozens of similar attempts. But realistic hope for oral insulin may finally be here. Click here to original publication. Fonte: SCIENCE sciencemag.org 8 FEBRUARY 2019 • VOL 363 ISSUE 6427 571…
Boom: 2018’s biotech IPOs
Biggest, richest, strongest, frothiest, riskiest; no matter what superlatives you want to apply, the biotech initial public offering (IPO) boom of 2018 was record breaking. In early December, Moderna Therapeutics’ US$604 million IPO — the largest ever for a venture-backed US biotech company — propelled the total capital raised in biotech IPOs during the year past the $8 billion threshold for the first time in the history of the sector… Click here to original publication. Fonte: NATURE REVIEWS | DRUG DISCOVERY VOLUME 18 | JANUARY 2019 | 3…
FDA approves landmark tissue-agnostic cancer drug
The FDA granted accelerated approval to Loxo Oncology and Bayer’s larotrectinib for patients with solid tumours that have neurotrophic receptor tyrosine kinase (NTRK) gene fusions. Larotrectinib is the first drug to be developed entirely for a tissue-agnostic cancer indication that doesn’t depend on where the cancer originated… Click here to original publication. Fonte: NATURE REVIEWS | DRUG DISCOVERY VOLUME 18 | JANUARY 2019 | 7…
Drug pipeline 3Q18
The third quarter saw a raft of first-in-class approvals, including the first RNA interference (RNAi) drug, the first nanobody, and a novel immunotoxin combining a CD22 Fv antibody fragment and an exotoxin. RNAi, antisense and small molecules achieved clinical… Fonte: VOLUME 36 NUMBER 12 DECEMBER 2018 NATURE BIOTECHNOLOGY Click here to original publication. …
Biopharmaceutical benchmarks 2018
Antibodies continue to dominate biopharmaceutical approvals, but new nucleic acid modalities and cellular therapies are also slowly launching on the market. This article provides an update on three previous surveys of biopharmaceutical approvals1–3. Fonte: VOLUME 36 NUMBER 12 DECEMBER 2018 NATURE BIOTECHNOLOGY Click here to original publication.…
First preventive mAb for hereditary angioedema
The US Food and Drug Administration has approved the first monoclonal antibody (mAb) to prevent hereditary angioedema (HEA) attacks. Dublin-based Shire was given the go-ahead in August to market Takhzyro (lanadelumab) as prophylactic treatment for HEA types I and II in patients aged 12 years or older. HEA is a rare but life-threatening genetic disease caused by mutations in the C1 esterase inhibitor gene, which leads to an overactivation of the complement system. This can trigger unpredictable bouts of subcutaneous or submucosal swelling anywhere in the body, potentially leading to blocked airways, and in…
FDA new drug approvals in Q3 2018
With one full quarter still to go before the end of the year, 41 novel drugs have already been approved by the FDA, putting 2018 on track to surpass 1996’s record 53 new drug approvals. Many of the 21 products approved in Q3 Fonte: NATURE REVIEWS | DRUG DISCOVERY VOLUME 17 | NOVEMBER 2018 | 779 Para acessar a publicação na íntegra clique aqui …
How much do phase III trials cost?
Drug development has been estimated to cost anywhere from US$43 million on one controversial end of the spectrum to $2.9 billion on the other when failures, post-approval studies and opportunity cost are factored in… Fonte: NATURE REVIEWS | DRUG DISCOVERY VOLUME 17 | NOVEMBER 2018 | 777 Click here to original publication.…
FDA approves first single-dose antimalarial
On July 20, the US Food and Drug Administration approved GlaxoSmithKline’s Krintafel (tafenoquine), a single-dose treatment to prevent relapses of Plasmodium vivax, a malaria parasite that causes more than eight million infections annually. “This drug could be transformative,” says David Reddy, executive director of Medicines for Malaria Venture, GlaxoSmithKline’s partner in the development of Krintafel. On August 9, the FDA also gave the go-ahead to 60 Degrees Pharmaceuticals for a six-month dosing regimen of tafenoquine tablets, commercialized as Arakoda, to prevent malaria in adults, including travelers and residents of disease-endemic countries… Click here to…
US approves first RNA interference drug
The first therapy based on RNA interference (RNAi) gene silencing has been approved by the US Food and Drug Administration (FDA). Alnylam Pharmaceuticals got the green light for Onpattro (patisiran), to treat hereditary transthyretin amyloidosis – a rare, progressive and often fatal disease. A mutation in patients with this hereditary disease makes a toxic form of transthyretin protein that is deposited in the heart and peripheral nerves. Patisiran silences this gene using short pieces of double-stranded RNA that target and…