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. Most, if not all, of these therapies used the FDA’s Orphan Drug Designation to aid their development plans.

Drugs 246

Drugs FDA Approval Marketing Development 246

Pharmaceutical Technology

SEPTEMBER 30, 2022

Skysona is indicated as a one-time gene therapy to slow the progression of cerebral adrenoleukodystrophy (CALD), a rare paediatric neurodegenerative disease in boys aged 4–17 years diagnosed with early-stage CALD. These approvals represent crucial milestones for bluebird bio, the gene therapy field, and patients with rare genetic diseases.

Gene Therapy 289

Gene Therapy FDA Approval Gene Genetic Disease 289

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.

Drugs 147

Drugs Medicine Marketing Development 147

XTalks

FEBRUARY 27, 2024

Rare Disease Day 2024, which falls on February 29 this year, is an opportunity to unite under a common cause: to bring attention to the challenges faced by those living with rare diseases and to push for advancements in research, treatment and policy. In 2020, 31 out of 53 novel drug approvals were for rare or orphan diseases​​.

Research 59

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. As covered in a previous article , the importance of R&D to develop treatments for rare diseases is high. of the global population are affected by a rare disease.

Drugs 52

Drugs Sales Marketing Development 52

XTalks

SEPTEMBER 27, 2022

CALD is a rare, progressive neurodegenerative disease that mainly affects young boys. The record-breaking price tag of Skysona is not surprising for one-time gene therapies like it. Skysona was granted Orphan Drug designation, Rare Pediatric Disease designation and Breakthrough Therapy designation. Related: Bluebird’s $2.8M

FDA Approval 52

FDA Approval Gene Therapy Drugs Gene 52

Delveinsight

FEBRUARY 9, 2021

Breyanzi will come with a price tag of USD 410,300 wholesale price, said the company, justifying it after taking into consideration several factors including medical and clinical value, patient value, and societal value. Ukoniq’s approval was backed by Phase 2 UNITY-NHL Trial (NCT02793583). The drug received priority review for the MZL.

FDA Approval 52

FDA Approval Marketing Research Protein 52