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. Efforts have been made to change this.

Drugs 147

Drugs Medicine Marketing Development 147

Pharmaceutical Technology

SEPTEMBER 30, 2022

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. bluebird then broke its own pricing record when the $3m Skysona was approved, and now faces the unique challenge of selling the world's two most expensive drugs.

Gene Therapy 289

Gene Therapy FDA Approval Gene Genetic Disease 289

XTalks

FEBRUARY 27, 2024

There have been significant advancements in new rare disease drugs, particularly for genetic disorders that can be treated by correcting, replacing or silencing defective genes. The US Food and Drug Administration (FDA) has approved a number of new rare disease drugs in recent years, including gene therapies.

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Research Drugs Gene Therapy Clinical Trials 59

pharmaphorum

JUNE 2, 2022

R&D into orphan drugs is growing alongside the number of approved treatments, providing treatments for rare diseases that previously did not have any. 141/2000 (the Orphan Regulation), that both acted to encourage development in the area, are regarded as crucial for changing the landscape in rare disease R&D.

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Drugs Sales Marketing Development 52

XTalks

SEPTEMBER 27, 2022

With a list price of $3 million and a recent accelerated US Food and Drug Administration (FDA) approval, bluebird bio’s gene therapy Skysona (elivaldogene autotemcel, eli-cel), has officially become the world’s most expensive drug. The record-breaking price tag of Skysona is not surprising for one-time gene therapies like it.

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FDA Approval Gene Therapy Drugs Gene 52

XTalks

MARCH 22, 2023

To celebrate this milestone, we’re taking a look back over the last 20 years in the pharmaceutical industry by highlighting some of the most talked-about mergers, blockbuster drugs, trends in FDA approvals and what’s next. In 2015, LabCorp acquired Covance , and Quintiles and IMS Health formed a joint venture in 2016, dubbed IQVIA.

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Life Science Drugs FDA Approval Vaccination 97

Delveinsight

FEBRUARY 9, 2021

The US Food and Drug Administration (FDA) has approved Bristol Myers Squibb’s (BMS) CAR T-cell therapy Breyanzi , for the treatment of adult patients with relapsed or refractory large B-cell lymphoma (LBCL) after two or more lines of systemic therapy, including Diffuse large B-cell lymphoma (DLBCL).

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FDA Approval Marketing Research Protein 52

FDA Law Blog

JUNE 27, 2023

Valentine Named Top Lawyer Under 40; Only Food and Drug Lawyer Selected Hyman, Phelps & McNamara, P.C. (HP&M) James was only one of five life sciences attorneys selected and the only food and drug lawyer to make the list. HP&M’s James E. Valentine , as a 2023 Rising Star.

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Life Science FDA Approval Drugs Development 81

Pharmaceutical Technology

JANUARY 27, 2023

On January 18, Sanofi launched a new warranty program for its rare blood disorder drug Cablivi (caplacizumab), which tackles value-based agreements in a slightly different way. Cablivi stops platelets from clumping together, which prevents small clots, and can be used along with other drugs that suppress the immune system.

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Manufacturing Drugs Research Analyst FDA Approval 130