Pharmaceutical Technology

APRIL 13, 2023

Two gene therapies up for approval this year for sickle cell disease could be cost effective in some cases at a $2 million price point, based on a draft evidence report published by the Institute for Clinical and Economic Review (ICER). Cell & Gene Therapy coverage on Pharmaceutical Technology is supported by Cytiva.

FDA 59

FDA 59

STAT

OCTOBER 6, 2022

… Multimillion-dollar prices for a rival’s treatments with the potential to cure rare diseases are “in the right ballpark,” according to Stuart Arbuckle, chief operating officer at Vertex Pharmaceuticals, which is preparing to market its own treatment for sickle-cell disease , Bloomberg News tells us. Even

98

98

pharmaphorum

JANUARY 11, 2021

Under the plans the company’s rare disease drugs will remain under the aegis of bluebird with current genetic disease president Andrew Obenshain taking the reins as CEO. million price tag. Meanwhile the as-yet unnamed oncology company will spin off under the leadership of bluebird’s current chief executive Nick Leschly.

FDA 66

FDA 66

pharmaphorum

JUNE 12, 2022

Cases of cancer associated with insertional mutagenesis, caused when the viral vectors used to deliver the gene therapy to host cells, have been seen with bluebird’s gene therapies but have been more apparent in trials involving its CALD and sickle cell disease candidates than beti-cel.

FDA 59

FDA 59

Pharmaceutical Technology

JUNE 29, 2022

This year has already been eventful when it comes to the development of therapies for rare diseases. Additionally, pricing and access for rare disease therapies continue to be scrutinized closely. Others judge its success based on the fact that 95% of rare diseases still have no available therapies and patient needs remain unmet.

Pharmaceutical Companies 98

Pharmaceutical Companies Pharmaceutical Companies FDA 98

pharmaphorum

FEBRUARY 22, 2021

Last week, Bluebird said it was pausing its Zynteglo (betibeglogene autotemcel) programme in Europe after a phase 1/2 trial in patients with sickle cell disease (SCD) – another red blood cell disorder – turned up two cases of blood cancers, one of acute myeloid leukaemia (AML) and another of myelodysplastic syndrome (MDS).

Immunization 52

Immunization 52

The Checkup by Singlecare

MARCH 21, 2023

Opioids should not be used as first-line therapy for short-term or chronic pain —with the exception being in end-of-life or palliative care, during active cancer treatment, and with sickle cell disease. Non-opioids should always be maximized before trialing opioids. RELATED: Belbuca side effects 2.

Dosage 52

Dosage Controlled Substances Labelling Packaging 52

The FDA Law Blog

JUNE 27, 2023

Amongst his accomplishments, Law360 considered the role James has played in leveraging little-used pathways to FDA approval for often first-ever drugs to treat rare diseases (e.g., Duchenne, Friederichs’s Ataxia, Sickle Cell Disease, Chagas, ALS). Dormer Jeffrey N. Gibbs Paul M. Hyman Alan M. Kirschenbaum Allyson B.

FDA 81

FDA 81

The Checkup by Singlecare

MAY 8, 2023

The Centers for Disease Control and Prevention (CDC) have committed to fighting the opioid overdose epidemic in many ways, including by aiding providers, healthcare systems, and payers with data, tools, and evidence-based guidance to support improved opioid prescribing and patient safety. fibromyalgia ).

Controlled Substances 52

Controlled Substances Dosage Labelling FDA 52

STAT

DECEMBER 15, 2023

… Bluebird Bio claimed a large insurer has agreed to cover its newly approved sickle cell disease gene therapy, easing some investor worries about resistance from other payers over the high price of the treatment, Reuters says. But Bluebird’s $3.1

Insurance 113

Insurance FDA 113