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Rare Disease Spotlight – tracing the rise of orphan drug designations over almost 40 years

Pharmaceutical Technology

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. Prior to the program, only 10 drugs were approved for a rare disease.

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PharmaShots Weekly Snapshots (May 01 - 05, 2023)

PharmaShots

Chiesi Global Rare Diseases and Protalix BioTherapeutics Receive EC’s Marketing Authorization of PRX-102 (pegunigalsidase alfa) for the Treatment of Fabry Disease Date: May 05, 2023 | Tags: Chiesi Global Rare Diseases, Protalix BioTherapeutics, PRX-102, pegunigalsidase alfa, Fabry Disease, Regulatory, EC, Marketing Authorization Kinoxis Therapeutics (..)

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PharmaShots Weekly Snapshots (May 22 - 26, 2023)

PharmaShots

Takeda and HUTCHMED's Fruquintinib Receives Priority Review from the US FDA to Treat Metastatic Colorectal Cancer Date: May 26, 2023 | Tags: Takeda, HUTCHMED, Fruquintinib, Metastatic Colorectal Cancer, Regulatory, Priority Review, US, FDA Gilead Receives EMA’s CHMP Positive Opinion to Extend the Use of Veklury (remdesivir) for COVID-19 Date: (..)

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PharmaShots Weekly Snapshots (May 08 - 12, 2023)

PharmaShots

Sanofi Reports P-IIIb Trial (HARMONIE) Results of Nirsevimab for the Prevention of Hospitalizations due to RSV-Related LRTD Date: May 12, 2023 | Tags: Sanofi, Nirsevimab, RSV-Related LRTD, Clinical Trial, P-IIIb, HARMONIE Trial G1 Therapeutics Presents Preliminary Results from P-II Trial of Trilaciclib for Triple-Negative Breast Cancer at ESMO 2023 (..)

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Benefits of blockchain in limiting counterfeit drug distribution

European Pharmaceutical Review

A review by Swedish researchers has identified the benefits of smart contracts on blockchain platforms in the pharmaceutical supply chain and its role in combating counterfeit drugs. The review stated that therapeutic innovation has resulted in the production of expensive drugs to treat rare diseases, which need to be available on time.

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Orphan drugs’ financial success raises questions

pharmaphorum

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.

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

Pharmaceutical Technology

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.