(Blog) New Cancer Drug, Three More for Rare Pediatric Diseases Approved, and a Voucher Sells for $95 Million

Thanksgiving week brought much gratitude from patient and advocate communities for four newly approved drugs for rare pediatric diseases, one of them for cancer. All four drugs earned FDA priority review vouchers under the program established by the Creating Hope Act in 2012 to stimulate drug development and provide incentives for companies to develop treatments for rare diseases in kids.

Danyelza (naxitamab), developed by  Y-mAbs Therapeutics, became the second drug for neuroblastoma and the third treatment for pediatric cancers approved under the Creating Hope Act in the past 8 years. It is indicated for pediatric patients one year of age and older and adult patients with relapsed or refractory high-risk neuroblastoma in bone or bone marrow.

Illustrating how the Creating Hope Act is meant to work — incentivizing drug companies to create drugs for rare diseases — Eiger BioPharmaceuticals developed a treatment for a disease that affects  about 20 kids in the U.S. The company received FDA approval for Zokinvy (lonafarnib) for the treatment of Hutchinson-Gilford Progeria Syndrome and processing-deficient Progeroid Laminopathies. A few days later Eiger sold its priority review voucher, earned for development of Zokinvy, to AbbVie for $95 million.

Rhythm Pharmaceuticals earned a voucher for the first-ever FDA approved therapy for rare genetic diseases of obesity. Imcirvee (setmelanotide) will be used for chronic weight management in adult and pediatric patients 6 years of age and older with obesity due to proopiomelanocortin (POMC), proprotein convertase subtilisin/kexin type 1 (PCSK1) or leptin receptor (LEPR) deficiency confirmed by genetic testing. 

And Alnylam Pharmaceuticals announced that it earned a rare pediatric disease voucher for Oxlumo (lumasiran), the first approved treatment for patients with primary hyperoxaluria type 1, which can cause kidney failure and become life threatening.

Since 2014, when the first drug earned a voucher under the Creating Hope Act, a total of 28 treatments for rare pediatric diseases have been  developed and received FDA rare pediatric disease priority review vouchers.