The Latest
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First look at CRISPR, Vertex gene-editing therapy hints at treatment potential
Results from two patients, one with sickle cell disease and the other with beta-thalassemia, offer an initial glimpse at the disease-altering potential of CRISPR gene-editing.
UPDATED: Nov. 19, 2019 at 8:38 a.m. -
SponsoredThe evolving demands of the temperature-controlled supply chain for cell and gene therapy
Cell and gene therapies are heightening the need for advanced temperature control solutions.
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France clears way for wide use of Vertex's Orkambi
By agreeing to reimburse the drug beyond compassionate use, France opens up access to one of the largest cystic fibrosis populations outside the U.S.
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Myovant to submit lead drug for prostate cancer approval after Phase 3 results
Next year will be a test of Myovant's commercial ambition, as the Roivant spin-off also plans to file relugolix for clearance in uterine fibroids.
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Dicerna links up with Novo in its 5th, likely final, liver-focused R&D deal
The Danish drugmaker will use the biotech's RNAi platform to develop therapies for diseases like NASH, diabetes and obesity.
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Alkermes follows up restructuring with Rodin acqusition
Worth up to $950 million, the deal will give Alkermes access to a preclinical pipeline aimed at diseases rooted in disrupted synapses like Alzheimer's and Parkinson's.
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Novartis reshapes China R&D hub to reflect rapidly evolving market
The Swiss pharma giant is ending drug discovery efforts and shifting resources toward early development and commercial activities, a move that will eliminate around 150 jobs in the near term.
UPDATED: Nov. 18, 2019 at 2:19 p.m. -
BeiGene FDA cancer drug nod seen as breakthrough for Chinese biopharma
The drug, branded as Brukinsa, will go up against J&J and AbbVie's Imbruvica and AstraZeneca's Calquence in lymphoma.
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Q&AWhat's next for Amarin? CEO John Thero talks Vascepa's label, pricing and future partnerships
Amarin will consider adding a partner in Europe, CEO John Thero told BioPharma Dive, adding he views the heart drug as a volume, not pricing, opportunity.
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Roche wagers $1.4B that Promedior has its next big fibrosis drug
Promedior's main drug targets a lung disease known as idiopathic pulmonary fibrosis and could help Roche once orphan exclusivity runs out on Esbriet.
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Novartis sickle cell drug wins FDA approval
Adakveo is only the second treatment approved in two decades for the painful blood clots caused by the rare disease.
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Sarepta brings in more gene therapies with StrideBio deal
The deal adds four more neuromuscular candidates to the Exondys maker's already-rich pipeline.
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ICER draws new gene therapy pricing framework
One-time and short-term curative therapies require a different approach to evaluating benefit, the cost watchdog group said.
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Rubius delays first clinical readout, as crowded PKU field marches on
With patient enrollment advancing more slowly than expected, the biotech pushed back the first look at human clinical data for its red blood cell therapeutic platform.
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Teva to restart production of cancer drug after shortages stressed treatment
Vincristine, a drug used to treat childhood cancers, has been in short supply since Teva stopped producing it and manufacturing woes hit Pfizer, the sole other supplier.
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FDA expert panel unanimously recommends Amarin's Vascepa in closely-watched vote
Debates about side effects and the mineral oil placebo failed to dissuade any member of the FDA's advisory committee from voting against an expanded indication.
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Lonza CEO Funk to step down after 9 months on job
Funk is leaving for personal reasons, Lonza reported in a brief statement, and will be replaced on an interim basis by chairman Albert Baehny.
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Merck wants to know if taking out cellular trash can treat Alzheimer's
The big pharma is acquiring Calporta Therapeutics to test whether drugs that promote lysosome function can combat neurodegenerative disorders.
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Sage's new drug starts slow, as Wall Street waits for depression data
Few expected Sage's postpartum depression drug Zulresso to start fast. Sales of $1.5 million in its first full quarter on market confirmed those expectations.
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Solid gene therapy trial halted again by FDA
Shares in Solid fell to a new low as the biotech's muscular dystrophy treatment dropped further behind the leader Sarepta Therapeutics.
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Reata's kidney data gets a tepid response from investors
Despite hitting the main goal of a Phase 3 study, Reata's drug showed that Alport syndrome patients must keep taking it to avoid further kidney decline.
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FDA documents appear to clear Amarin path to wider heart drug approval
The documents, released ahead of Thursday's advisory panel meeting, suggest the FDA is open to an expanded label for Vascepa, Amarin's fish oil-derived pill.
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Regenxbio, stung by trial hold on gene therapy, sues FDA
The biotech seeks to overturn clinical holds placed by the FDA on two gene therapy programs, arguing the agency action was "arbitrary and capricious."
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Roche's oral SMA drug scores in older patients
As Novartis' gene therapy Zolgensma remains under scrutiny, the Swiss rival's risdiplam delivered positive results in older, less severely affected patients.
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Novartis' Sandoz builds global generics presence with Japan acquisition
Sandoz will pay approximately $331 million upfront for a Japan-focused subsidiary of Aspen Pharmacare.
