NULIBRY (fosdenopterin hydrobromide)

The pharmacokinetics of fosdenopterin in healthy adult subjects following a single intravenous NULIBRY infusion are summarized in Table 3 . The area under the plasma concentration-time curve (AUC) and the maximum plasma concentration (C max ) of fosdenopterin increased in an approximately proportional manner with increasing doses.

Table 3Mean (SD) Pharmacokinetic Parameters Following A Single Intravenous Dose of Fosdenopterin in Healthy Subjects 1 0.075 mg/kg, 0.24 mg/kg, and 0.68 mg/kg doses a...

The efficacy of NULIBRY for the treatment of patients with MoCD Type A was established based on data from three clinical studies (Studies 1, 2, and 3) that were compared to data from a natural history study.

Study 1

Study 1(NCT02047461) was a prospective, open-label, single-arm, dose escalation study in patients with MoCD Type A who were receiving treatment with rcPMP prior to treatment with NULIBRY. Study 1 included 8 patients, 6 of whom previously participated in Study 3. The initial NULIBRY dosage was matched to the patient's rcPMP dosage upon entering the study. The NULIBRY dosage was the...

The most common adverse reactions (>25%) were catheter-related complications, pyrexia, viral infection, pneumonia, otitis media, vomiting, cough/sneezing, viral upper respiratory infection, gastroenteritis, bacteremia, and diarrhea. ( 6.1 ) To report SUSPECTED ADVERSE REACTIONS, contact Sentynl Therapeutcis, Inc. at 888-507-5206 or FDA at 1-800-FDA-1088 or www.fda.gov/medwatch. 6.1 Clinical Trials Experience Because clinical trials are conducted under widely varying conditions, adverse reaction ...

None. None. ( 4 )

Potential for Photosensitivity : Advise patients/caregivers to avoid patient exposure to sunlight, and to have the patient wear sunscreen, protective clothing, and sunglasses when exposed to the sun. If photosensitivity occurs, advise caregivers/patients to seek medical attention immediately and consider a dermatological evaluation. ( 5.1 , 13.2 ) 5.1 Potential for Photosensitivity Animal studies have identified that NULIBRY has phototoxic potential [see Nonclinical Toxicology ( 13.2 )]. Advise ...