August Advances in Rare Disease Drug Development: Key Approvals and Pipeline Highlights

Sep 03, 2024 20:00 CST Updated 20:00

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Advances in Rare Disease Drug Development

☞ Takeda's New Drug for Von Willebrand Disease Approved in ChinaListed

☞ Changchun Jin Sai's long-acting growth hormone approved for new indications in China

☞ AstraZeneca's Long-Acting Enzyme Replacement Therapy Successfully Introduced in the Greater Bay Area

☞ Gilead's New Drug for Primary Biliary Cholangitis Approved and Launched in the United States

☞ Roche's PNH Antibody Therapy Approved for Marketing in the EU

Takeda's New Drug for Von Willebrand Disease Approved for Marketing in China

Takeda announced that its recombinant von Willebrand factor (rVWF) injection, Vonicog alfa (brand name: Veinaze®), has received marketing approval from the NMPA for on-demand treatment and bleeding episode control in adult patients with von Willebrand disease (VWD), as well as perioperative bleeding management.

Injectable Vonicog Alfa is a recombinant VWF factor produced using genetic recombination technology. It contains full-length VWF multimers, including ultra-large multimers, and has a longer half-life, enabling replacement therapy for von Willebrand disease.

Changchun Jin Sai's Long-Acting Growth Hormone Receives New Indication Approval in China

The NMPA official website shows that Changchun Jin Sai Pharmaceutical's polyethylene glycol recombinant human growth hormone injection (trade name: Jin Sai Zeng) has been approved for a new indication, used to treat growth disorders in children caused by Turner syndrome.

Jin Sai Zeng is a long-acting growth hormone that requires only one injection per week. It was first approved for marketing in China in 2014 for the treatment of slow growth in children caused by endogenous growth hormone deficiency; in 2023, it was approved for a new indication for the treatment of growth disorders in adolescents caused by chronic kidney disease.

AstraZeneca's Long-Acting Enzyme Replacement Therapy Successfully Introduced in the Greater Bay Area

AstraZeneca announced that its developed long-acting enzyme replacement therapy, Strensiq (asfotase alfa injection), has been approved by the Guangdong Provincial Drug Administration and officially introduced to the medical institutions designated by the Greater Bay Area drug and device program for the treatment of infantile-onset hypophosphatasia (HPP).

Asfotase Alfa can replace the alkaline phosphatase lacking in patients and serve as a long-term enzyme replacement therapy to control patient symptoms. Clinical data shows that Asfotase Alfa injection significantly improves patient survival rates: the survival rate of patients treated with Asfotase Alfa injection is 95% at 1 year old, 86% at 3.5 years old, and 82% at 5 years old.

Gilead's New Drug for Primary Biliary Cholangitis Approved for Marketing in the United States

Gilead announced that its self-developed Livdelzi (seladelpar) has received accelerated approval from the FDA for use in combination with ursodeoxycholic acid (UDCA) to treat adult patients with primary biliary cholangitis (PBC) who have an inadequate response to UDCA, or as a monotherapy for patients who cannot tolerate UDCA. Livdelzi is an orally administered potent and selective peroxisome proliferator-activated receptor δ (PPARδ) agonist, which can participate in bile acid synthesis, inflammation, fibrosis, and lipid metabolism.

Roche's PNH Antibody Therapy Approved for Marketing in the EU

Roche announced that its developed C5 antibody PiaSky® (crovalimab) has been approved by the EMA for marketing, indicated for the treatment of paroxysmal nocturnal hemoglobinuria (PNH) in adult and adolescent patients aged 12 years and older (weighing 40 kg or more) who have either not received or have previously received C5 inhibitor therapy.

PiaSky is a humanized monoclonal antibody that requires only one subcutaneous injection per month. It is designed to bind to complement C5, blocking the complement pathway of the immune system to achieve therapeutic effects. Through engineering modifications using continuous monoclonal antibody recycling technology, PiaSky can rapidly and durably inhibit the complement pathway at a lower dose.

Source| Shanghai Pharma Rui'er

Editor | Subi

Rare Neurological Disorders Communication Group

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