Pharmacist Digest

STAT+: Kyowa Kirin to acquire Orchard Therapeutics, maker of gene therapy Libmeldy

STAT

OCTOBER 5, 2023

The medicines, which deliver functional genes to patients to overcome their genetic diseases, are one-time treatments that can have profound effects for patients but that typically carry seven-figure price tags, leading to so far slow rollouts. Continue to STAT+ to read the full story…

STAT+: Pharmalittle: Some employers cut insurance coverage for weight loss drugs; U.S. urges ADHD pill makers to boost production

STAT

AUGUST 2, 2023

… So many people have turned to drugs used for weight loss that some employers are cutting off insurance coverage to head off climbing bills , The Wall Street Journal reports. The outlays are straining the finances of some plans, including those funded by employers. Besides the $26,500 annual price tag, treatment could cost U.S.

bluebird bio wins back-to-back landmark FDA approvals for first-in-class gene therapies

Pharmaceutical Technology

SEPTEMBER 30, 2022

These approvals represent crucial milestones for bluebird bio, the gene therapy field, and patients with rare genetic diseases. Spark Therapeutics’ Luxturna, indicated for inherited retinal disease (IRD), was the first gene therapy to be approved, in 2017, with a price tag of $850,000 for each eye.

Free access to Zolgensma curbed, says Novartis

pharmaphorum

JANUARY 9, 2023

First introduced in 2020, the global Managed Access Programme (gMAP) has provided Zolgensma (onasemnogene abeparvovec) free of charge to nearly 300 children with the genetic disorder across 36 countries where the therapy has not yet received approval or in which no formal access pathway exists.

Cartherics grants licence for CTH-004 to Shunxi

Pharmaceutical Technology

MAY 1, 2023

CTH-004 is developed by genetically altering patient T cells for inserting a chimeric antigen receptor (CAR) to target a marker (TAG-72) on ovarian cancer cells and delete genes which are involved in T cell function suppression. Topic sponsors are not involved in the creation of editorial content.

NICE reaches a deal with Roche on access to oral SMA drug Evrysdi

pharmaphorum

NOVEMBER 19, 2021

Roche’s oral treatment Evrysdi for the rare genetic disease spinal muscular atrophy (SMA) will be made available on the NHS in England, after NICE reached a three-year access agreement with the company. million price tag make it is the most expensive treatment ever approved for NHS funding.

A broad range of unmet needs remains in the immuno-oncology space

Pharmaceutical Technology

MAY 2, 2023

There are more than 20 marketed ICIs with approvals across a very wide spectrum of solid tumour indications. In contrast to ICIs, which are utilised against solid tumours, cell therapies and bispecifics have been transformational in the haematological cancer settings, with approvals across a range of leukaemias and lymphomas.

Immunity

Immunity Immunization Vaccines

After a slow start for expensive CAR-T therapies, drug developers revisit oral therapies for blood cancer

pharmaphorum

DECEMBER 23, 2022

But along with strong efficacy come some considerable risks and costs: cytokine release syndrome (CRS), where a patient’s immune system becomes dangerously over-stimulated, and neurotoxicity are potential side effects that need to be managed. These are particularly important in the progression of certain blood cancers.

Drug Development

Drug Development Immunity Immunization Hospitals

Orphan drugs and where to launch them: The keys to Europe’s forgotten territories

Pharmaceutical Technology

JULY 14, 2022

It is estimated that there are currently more than 7,000 orphan diseases, many of which are considered life-threatening and most of which have a genetic basis. Despite this high number, orphan diseases are rare by definition, affecting around one in 2,000 people as defined by the European Union. Go-to-market strategies.

Pharmaceutical Companies

Pharmaceutical Companies Pharmaceutical Companies Hospitals

bluebird unveils $2.8m price for gene therapy Zynteglo on FDA approval

pharmaphorum

AUGUST 18, 2022

Zynteglo is a customised, one-time treatment created using a patient’s own bone marrow stem cells hat are genetically modified to produce functional beta-globin, which is mutated in the disease. million price tag attached to the therapy when it was first made available in Europe, and is also well above the proposed $2.1

Zynteglo halt re-ignites viral vector safety concerns; analysts

pharmaphorum

FEBRUARY 22, 2021

If a causal link is shown, it could have “significant ramifications” on the use of lentiviral vectors, a type of self-inactivating virus that underpins both approved cell therapies like Novartis’ CAR-T Kymriah as well as a host of experimental therapies that involve genetic manipulation of cells. million in Europe.

Immunity

Immunity Immunization

NICE ‘no’ to Roche’s risdiplam for SMA not unexpected, says patient group

pharmaphorum

JUNE 2, 2021

The cost-effectiveness agency’s initial assessment is that Evrysdi is simply too expensive at its current price to be provided to the roughly 1,500 people with the rare genetic disorder who might be eligible to receive it. Zolgensma was backed for type 1 SMA in draft guidance issued last month, even though the gene therapy’s £1.79

Documentation

Documentation Communication

Q&A: A decade on, what’s next for CAR-T therapies?

Pharmaceutical Technology

APRIL 5, 2023

But access to these treatments continues to remain limited due to high price tags and variable availability across regions. Currently, all approved CAR-T therapies are autologous, where T cells taken from patients are modified and then re-infused. There are issues of geography, but also issues of economics.

Immunity

Immunity Immunization

Realising milestones with gene therapy for SMA

European Pharmaceutical Review

MAY 11, 2023

Three SMN-enhancing treatments for the condition are approved by the US Food and Drug Administration (FDA) 2 , however there is no cure for SMA at present. There are three modes of action (MOA) available for treatments – intrathecal injection, intravenous infusion and an oral formula. percent of Europe’s annual healthcare spend.

Drug Development

Twenty Women Leaders In Healthcare Industry

PharmaShots

MARCH 9, 2023

We are esteemed and elated to present a list of the 20 women who have excelled in reaching the summit of excellence, arranged alphabetically Total experience: 16 yrs. Other Affiliations: She holds New York State Department of Health certification in Molecular Genetics and Molecular Oncology. LinkedIn Total experience: 27 yrs.

Clinical Pharmacology

Clinical Pharmacology Immunity Immunization Pharmaceutical Companies

Putting complex medicines under the microscope

European Pharmaceutical Review

NOVEMBER 10, 2022

Advanced light microscopy techniques are one tool that can be used to understand these medicines. While there is a significant market opportunity in this sector, advanced analytical methods are required, and many technologies cannot provide the resolution needed. What constitutes a complex medicine? Physical characterisation.

Labelling

Labelling Compounding

Viagra ‘should be tested as Alzheimer’s therapy’

pharmaphorum

DECEMBER 7, 2021

They used computational techniques to sift through genetic and other biologic data as well as insurance claims information from a database of more than 7 million patients, and found that sildenafil had the greatest potential as an Alzheimer’s drug.

Compounding

Compounding Diabetes

Developing point-of-care CAR T manufacturing

European Pharmaceutical Review

DECEMBER 27, 2023

Chimeric antigen receptor (CAR) T cells are the first example of a “living drug”. 1 Following in vitro production from T cells collected from a patient’s leukapheresis procedure, the CAR T cells are infused back into the patient’s blood, where they proliferate and expand. This is in addition to its considerable price tag.

Adverse Reactions

Adverse Reactions Immunity Immunization Medical Schools

Pharmacist Digest

STAT+: Kyowa Kirin to acquire Orchard Therapeutics, maker of gene therapy Libmeldy

STAT+: Pharmalittle: Some employers cut insurance coverage for weight loss drugs; U.S. urges ADHD pill makers to boost production

Trending Sources

bluebird bio wins back-to-back landmark FDA approvals for first-in-class gene therapies

Free access to Zolgensma curbed, says Novartis

Cartherics grants licence for CTH-004 to Shunxi

NICE reaches a deal with Roche on access to oral SMA drug Evrysdi

A broad range of unmet needs remains in the immuno-oncology space

After a slow start for expensive CAR-T therapies, drug developers revisit oral therapies for blood cancer

Orphan drugs and where to launch them: The keys to Europe’s forgotten territories

bluebird unveils $2.8m price for gene therapy Zynteglo on FDA approval

Zynteglo halt re-ignites viral vector safety concerns; analysts

NICE ‘no’ to Roche’s risdiplam for SMA not unexpected, says patient group

Q&A: A decade on, what’s next for CAR-T therapies?

Realising milestones with gene therapy for SMA

Twenty Women Leaders In Healthcare Industry

Putting complex medicines under the microscope

Viagra ‘should be tested as Alzheimer’s therapy’

Developing point-of-care CAR T manufacturing

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