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.

Pharmaceutical Companies 59

Pharmaceutical Companies Pharmaceutical Companies Hospitals 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.

Compounding 52

Compounding Pharmaceutical Companies Pharmaceutical Companies 52

PharmaShots

JANUARY 27, 2023

Magenta Therapeutics Pauses the P-I/II Study in AML Patients Date: Jan 27, 2023 | Tags: Magenta Therapeutics, MGTA-117, AML, Clinical Trial, P-I/II Ipsen Receives CHMP Negative Opinion for Palovarotene to Treat Fibrodysplasia Ossificans Progressiva Date: Jan 27, 2023 | Tags: Ipsen, Palovarotene, Fibrodysplasia Ossificans Progressiva, Regulatory, CHMP, (..)

FDA 40

FDA Immunization 40

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. The disorder has a reported annual incidence between 1.5 cases per million in Europe. Currently, Cablivi’s WAC is $7,300 per single-dose vial kit.

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Documentation Immunization Pharmaceutical Companies Pharmaceutical Companies 52