pharmaphorum

AUGUST 21, 2022

Shares in Axsome Therapeutics have rocketed on FDA approval of its depression therapy Auvelity (formerly AXS-05) – a year after its approval was held up by the regulator. In this product, bupropion is designed to increase levels of dextromethorphan in the blood and extend its half-life.

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FDA Marketing Pharma Sales 88

pharmaphorum

AUGUST 25, 2022

Johnson & Johnson has its first worldwide regulatory approval – in the EU – for Tecvayli, one of two much-anticipated bispecific antibodies the drugmaker has developed for patients with multiple myeloma. Tecvayli previously scored a priority medicines (PRiME) designation from the EMA and a breakthrough-therapy tag from the FDA.

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FDA Marketing Safety Patients 93

pharmaphorum

OCTOBER 3, 2022

Amylyx Pharmaceuticals’ Relyvrio – a new treatment for amyotrophic lateral sclerosis (ALS) approved by the FDA on Thursday, after review of the data from its phase 2 trial – was the next day set at a list price of $158,000 per year in the US, sparking outcry. ALS affects over 30,000 people in the US.

Insurance 52

Insurance Prescription FDA Patients 52

Pharmaceutical Technology

NOVEMBER 9, 2022

An approval decision on the gene therapy, also known as EtranaDez, is expected by the end of this month. On November 7, the company announced that the FDA has scheduled a pre-licensure inspection of BioMarin’s manufacturing facility, and required the company to submit three-year Phase III Roctavian data in the approval application.

Safety 111

Safety Pharmaceutical Patients Medicine 111

pharmaphorum

AUGUST 18, 2022

bluebird bio’s Zynteglo has become the first cell-based gene therapy to be approved in the US, getting the nod from the FDA as a treatment for patients with beta thalassaemia who require regular blood transfusions. An FDA advisory committee recommended approval of Zynteglo on the strength of that data in June.

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Pharmaceutical Technology

JUNE 29, 2022

The US Food and Drug Administration (FDA) put a high-profile bluebird bio trial for sickle cell disease on partial clinical hold, and advisory panels deliberated over decisions involving gene therapies for amyotrophic lateral sclerosis (ALS), cerebral adrenoleukodystrophy (CALD), and beta-thalassemia.

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Food and Drug Administration FDA Marketing Pharmaceutical 98

pharmaphorum

JULY 25, 2022

The EMA’s human medicines committee has recommended approval of Johnson & Johnson’s Tecvayli as a fourth-line therapy for multiple myeloma , joining a growing group of BCMA-targeted therapies for the blood cancer.

Medicine 52

Medicine FDA Recruitment Patients 52

Pharmaceutical Technology

FEBRUARY 10, 2023

On November 22, 2022, the FDA approved CSL Behring’s Hemgenix (etranacogene dezaparvovec), the first gene therapy treatment for hemophilia B, with a staggering manufacturer price of $3.5 This is not the first treatment to come with a high price tag.

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Marketing Medicine Leads Safety 105

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). If approved, exa-cel would be the first FDA-approved gene therapy based on CRISPR editing.

Healthcare 59

Healthcare Healthcare Pharmaceutical FDA 59

pharmaphorum

DECEMBER 12, 2022

Vyvgart is already approved to treat generalised myasthenia gravis, getting a green light for that indication from the FDA a year ago, and launched with a $225,000 per annum price tag in competition with rival gMG drugs like AstraZeneca/Alexion’s C5 inhibitor Soliris (eculizumab).

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Side effects Sales Competition Patients 52

World of DTC Marketing

DECEMBER 13, 2021

As cancer survival rates rise, so do the price tags of life-saving treatments. 11 of the 12 cancer drugs that the Food and Drug Administration approved in 2012 were priced at more than $100,000 per year. OPENING: American cancer patients spent more than $21 billion on their care in 2019. billion included out-of-pocket costs of $16.22

Food and Drug Administration 55

Food and Drug Administration Food Medical Insurance 55

Pharmaceutical Technology

APRIL 5, 2023

More than a decade after the first patient was treated with a CAR-T therapy, six therapies relying on the same principles have been approved by the US Food and Drug Administration (FDA) and marketed to thousands of patients. It will be six years since the US FDA approval of Kymriah in August this year.

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Food and Drug Administration Engineering Patients Education 59

Pharmaceutical Technology

FEBRUARY 22, 2023

The legal dispute is centered around rival cholesterol-lowering drugs produced by the two companies, which were approved in the summer of 2015. Developed by Sanofi and Regeneron, Praluent was approved by the FDA in July 2015. Both drugs come with a high price tag. Amgen’s Repatha was greenlit a month later.

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Pharma Government FDA Sales 104

PM360

OCTOBER 28, 2022

First, as every pharma marketer knows, Medical Legal Regulatory (MLR) review and approval is a legal layer of consumer protection, confidence, and clarity. Current processes used by many pharma companies include building campaigns with bespoke coding and duplicative review and approval chains.

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Marketing Pharma Consulting Management 98

pharmaphorum

AUGUST 26, 2022

BioMarin Pharma has its landmark first approval for Roctavian – the first for a haemophilia gene therapy – and will now have to see if that can convert that into a viable business in Europe. Meanwhile, BioMarin has also said it intends to refile for approval of Roctavian in the US before the end of next month.

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FDA Marketing Competition Patients 108

Pharmaceutical Technology

JUNE 21, 2023

CSL announced the first patient with haemophilia B has been treated with its recently approved gene therapy Hemgenix (etranacogene dezaparvovec). Hemgenix is the first and only gene therapy approved for haemophilia B. Despite the limited market, the price tags of haemophilia make it potentially lucrative.

Patients 98

Patients Food and Drug Administration Engineering Sales 98

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. While Zolgensma is also being investigated in the form of an injection, the MOA currently approved by the FDA is a single infusion.

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Food and Drug Administration Government Healthcare Healthcare 75

Pharmaceutical Technology

JANUARY 27, 2023

In 2019, Cablivi became the first FDA-approved , nanobody-based medicine for acquired thrombotic thrombocytopenic purpura (aTTP), an ultra-orphan condition that causes blood clots in small blood vessels around the body. In 2022, the number of risk sharing agreements peaked with 144 agreements being made in the pharmaceutical industry.

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Patients Pharmaceutical Manufacturing Healthcare Provider 52

PM360

SEPTEMBER 7, 2023

Mason learned that lesson while working with argenx to launch VYVGART (efgartigimod alfa-fcab), the first FDA-approved treatment of its kind for adults with anti-AChR antibody positive generalized myasthenia gravis (gMG), a rare autoimmune neuromuscular disease. We also put big pull tags on the little pouches that held the items.

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Patients Pharma Education Doctors 105

European Pharmaceutical Review

DECEMBER 27, 2023

Currently CAR T-cell therapy is approved for three indications: acute lymphoblastic leukaemia (ALL), non-hodgkin lymphoma (NHL), and multiple myeloma (MM) – and the strongest correlation between CAR T-cell efficacy and persistence has been demonstrated in ALL. This is in addition to its considerable price tag. 2021;3(S1):6–10.

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Manufacturing Food and Drug Administration Patients Pharma 89

Pharmaceutical Technology

NOVEMBER 30, 2022

In October, the US FDA granted Eli Lilly’s Mounjaro (tirzepatide) a Fast Track designation for its use as a treatment for obesity. The drug was approved to treat type 2 diabetes mellitus in May. In the first quarter since its launch as an approved therapy for type 2 diabetes, Mounjaro has proven to be a lucrative drug, making $97.3

Medical 105

Medical Manufacturing Insurance Side effects 105

Pharmaceutical Technology

NOVEMBER 30, 2022

In October, the US FDA granted Eli Lilly’s Mounjaro (tirzepatide) a Fast Track designation for its use as a treatment for obesity. The drug was approved to treat type 2 diabetes mellitus in May. In the first quarter since its launch as an approved therapy for type 2 diabetes, Mounjaro has proven to be a lucrative drug, making $97.3

Medical 98

Medical Manufacturing Insurance Side effects 98