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Pretomanid

Generic Name
Pretomanid
Brand Names
普瑞尼, Dovprela (previously Pretomanid FGK)
Drug Type
Small Molecule
Chemical Formula
C14H12F3N3O5
CAS Number
187235-37-6
Unique Ingredient Identifier
2XOI31YC4N
Background

Persistent forms of tuberculosis (TB) have proven to be a major cause of global morbidity and mortality and a cause for significant concern. Research in recent years has been geared toward the development of novel therapies that target persistent forms of this disease, which have shown resistance to standard therapy regimens. Pretomanid is an antimycobacterial agent that is administered with Bedaquiline and Linezolid to treat resistant forms of pulmonary TB. It was the first TB drug developed by a nonprofit organization, known as TB Alliance, and was granted FDA approval on August 14, 2019. Unlike other therapeutic regimens for the treatment of resistant TB, which may take 18 months or longer and may not be effective, the pretomanid-containing regimen allows for a more efficacious and shorter duration of treatment with fewer drugs.

Indication

Pretomanid is indicated, as part of a combination regimen with bedaquiline and linezolid, for the treatment of adults with pulmonary tuberculosis (TB) that is resistant to isoniazid, rifamycins, a fluoroquinolone and a second-line injectable antibacterial drug or adults with pulmonary TB resistant to isoniazid and rifampin, who are treatment-intolerant or non-responsive to standard therapy.

Associated Conditions
Pulmonary Rifampicin- and Isoniazid-Resistant Tuberculosis, Pulmonary Tuberculosis resistant to isoniazid, rifamycins, a fluoroquinolone and a second line injectable antibacterial drug

Congress's Failure to Renew Priority Review Voucher Program Threatens Rare Disease Drug Development

• The FDA's rare pediatric disease Priority Review Voucher program expired in December 2023, despite unanimous congressional support for renewal, leaving biotech companies unable to receive new vouchers worth approximately $150 million. • Eleven priority review vouchers were awarded in 2023, marking a record year, with beneficiaries including treatments for sickle cell disease, Duchenne muscular dystrophy, and other rare conditions. • The program's suspension threatens future rare disease drug development, as many companies rely on voucher sales for funding, with existing designations only valid until September 2026 for voucher eligibility.
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