Another Priority Review Voucher was born, who is the last "oriole"

In March 2022, the U.

Ztalmy: Seizures associated with cyclin-dependent kinase-like 5 (CDKL5) deficiency (CDD) in patients 2 years of age and older;

Opdualag: unresectable or metastatic melanoma in adults and pediatric patients 12 years of age and older;

Hyftor: angiofibroma of the face associated with tuberous sclerosis complex in adults and pediatric patients 6 years of age and older;

Xelstrym: Attention Deficit Hyperactivity Disorder (ADHD) in adults and pediatric patients 6 years of age and older;

Triumeq PD: HIV-1 infection in pediatric patients 10-25 kg;

2 FDA-approved indications for new medicines for children susceptible to diseases:

Atropine Sulfate: mydriasis, cycloplegia, and depression of the dominant eye in the treatment of amblyopia;

Nasonex 24hr Allergy: Temporary relief of hay fever or other upper respiratory allergy symptoms: stuffy nose, runny nose, sneezing, itchy nose;

01

FDA Legislation Forces and Incentivizes Children's Drug Development

Children are not little adults.

In addition to encouraging and mandatory requirements, the United States has established a very attractive incentive policy for children's drug research and development: the Pediatric Orphan Drug Priority Review Voucher (PRV)

In February 2017, Sarepta, a company that treats Duchenne muscular dystrophy, sold the pediatric rare drug priority review ticket it obtained to Gilead, and the transaction price has doubled to $125 million

Among the 13 new drugs approved by the FDA this time, another PRV was born.

02

Lucky PRV, involving a new drug for rare hereditary epilepsy!

MarinusPharma is a pharmaceutical company dedicated to developing innovative treatments for epilepsy

CDKL5 deficiency (CDD) is a severe and rare inherited form of epilepsy characterized by early onset, uncontrolled seizures, and severely impaired neurodevelopment

It is worth mentioning that Ztalmy is the first FDA-approved drug specifically for the treatment of CDD

Chemical structure of ganaxolone (Image source: Wikipedia.

Ztalmy's active pharmaceutical ingredient is ganaxolone, a positive allosteric modulator of GABAA receptors, currently in development in intravenous and oral formulations designed to maximize treatment for adult and pediatric patient populations in acute and chronic care settings range

The FDA approves Ztalmy for the treatment of CDD-related seizures, based on data from the Phase 3 Marigold trial

In the Marigold open-label extension study, patients treated with Ztalmy for at least 12 months (n=48) had a median reduction of 49.

Ztalmy was approved through a priority review process
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Previously, the FDA has granted Ztalmy Orphan Drug Designation (ODD) and Rare Pediatric Disease Designation (RPDD) for the treatment of CDD
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With this approval, the FDA issued a Pediatric Orphan Drug Priority Review Voucher (PRV) to Marinus, which the company plans to sell for cash
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"The mantis catches the cicada, the oriole is behind", we will wait and see who this PRV will end up with
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summary

For a long time, due to the relatively early progress of scientific research in the United States and the relatively developed scientific research, the FDA's new drug review and approval has also been at the forefront of drug research and development approvals in various countries
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To some extent, FDA's systems and preferences influence the direction of new drug development on a global scale
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The FDA has approved a number of new drugs that can be used for children, reflecting the FDA's emphasis on and prioritizing the review and approval of children's drugs, or affecting subsequent drug research and development and enterprise layout
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After years of development, domestic drug research and development has achieved small achievements and is catching up quickly
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Domestic pharmaceutical companies should pay attention to the weather vane revealed by the FDA's drug approval, and then revise and decide the strategic layout of the company after comprehensive consideration, so as to win a seat in the international competition
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Relying on a powerful data and information platform, the China Pharmaceutical Industry Information Center has collected and sorted out the fourth phase of the new global pharmaceutical research in 2022, empowering enterprise R&D through big data, reducing hidden dangers caused by information asymmetry, and formulating patent layouts more efficiently And product development strategy, hoping to have an important reference for the establishment of research and development projects of pharmaceutical companies
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