Nulibry

Nulibry Uses, Dosage, Side Effects, Food Interaction and all others data.

Molybdenum cofactor deficiency (MoCD) is an exceptionally rare autosomal recessive disorder resulting in a deficiency of three molybdenum-dependent enzymes: sulfite oxidase (SOX), xanthine dehydrogenase, and aldehyde oxidase. Signs and symptoms begin shortly after birth and are caused by a build-up of toxic sulfites resulting from a lack of SOX activity. Patients with MoCD may present with metabolic acidosis, intracranial hemorrhage, feeding difficulties, and significant neurological symptoms such as muscle hyper- and hypotonia, intractable seizures, spastic paraplegia, myoclonus, and opisthotonus. In addition, patients with MoCD are often born with morphologic evidence of the disorder such as microcephaly, cerebral atrophy/hypodensity, dilated ventricles, and ocular abnormalities. MoCD is incurable and median survival in untreated patients is approximately 36 months - treatment, then, is focused on improving survival and maintaining neurological function.

The most common subtype of MoCD, type A, involves mutations in MOCS1 wherein the first step of molybdenum cofactor synthesis - the conversion of guanosine triphosphate into cyclic pyranopterin monophosphate (cPMP) - is interrupted. In the past, management strategies for this disorder involved symptomatic and supportive treatment, though efforts were made to develop a suitable exogenous replacement for the missing cPMP. In 2009 a recombinant, E. coli-produced cPMP was granted orphan drug designation by the FDA, becoming the first therapeutic option for patients with MoCD type A.

Nulibry was approved by the FDA on Februrary 26, 2021, for the reduction of mortality in patients with MoCD type A, becoming the first and only therapy approved for the treatment of MoCD. By improving the three-year survival rate from 55% to 84%, and considering the lack of alternative therapies available, fosdenopterin appears poised to become a standard of therapy in the management of this debilitating disorder.

Nulibry
Uses
Dosage
Side Effect
Precautions
Interactions
Uses during Pregnancy
Uses during Breastfeeding
Accute Overdose
Food Interaction
Half Life
Volume of Distribution
Clearance
Interaction With other Medicine
Contradiction
Storage

Trade Name	Nulibry
Availability	Prescription only
Generic	Fosdenopterin
Fosdenopterin Other Names	CPMP, Fosdenopterin
Related Drugs	Nulibry
Weight	9.5mg,
Type	Intravenous powder for injection
Formula	C₁₀H₁₄N₅O₈P
Weight	Average: 363.223 Monoisotopic: 363.057999429
Protein binding	Plasma protein binding ranges from 6 to 12%, though the specific proteins to which fosdenopterin binds have not been elucidated.
Groups	Approved
Therapeutic Class
Manufacturer
Available Country	United States,
Last Updated:	September 19, 2023 at 7:00 am

Uses

Nulibry is an exogenous form of cyclic pyranopterin monophosphate (cPMP) used as a replacement substrate in patients with molybdenum cofactor deficiency (MoCD) type A.

Nulibry is indicated to reduce the risk of mortality in patients with molybdenum cofactor deficiency (MoCD) type A.

Nulibry is also used to associated treatment for these conditions: Molybdenum Cofactor Deficiency, Type A

How Nulibry works

Molybdenum cofactor deficiency (MoCD) is a rare autosomal-recessive disorder in which patients are deficient in three molybdenum-dependent enzymes: sulfite oxidase (SOX), xanthine dehydrogenase, and aldehyde dehydrogenase. The loss of SOX activity appears to be the main driver of MoCD morbidity and mortality, as the build-up of neurotoxic sulfites typically processed by SOX results in rapid and progressive neurological damage. In MoCD type A, the disorder results from a mutation in the MOCS1 gene leading to deficient production of MOCS1A/B, a protein that is responsible for the first step in the synthesis of molybdenum cofactor: the conversion of guanosine triphosphate into cyclic pyranopterin monophosphate (cPMP).

Nulibry is an exogenous form of cPMP, replacing endogenous production and allowing for the synthesis of molybdenum cofactor to proceed.

Toxicity

There are no data regarding overdosage of fosdenopterin.

Food Interaction

No interactions found.

Volume of Distribution

The volume of distribution of fosdenopterin is approximately 300 mL/kg.

Elimination Route

In healthy adult subjects, the observed C_max and AUC_0-inf following the intravenous administration of 0.68 mg/kg (0.76x the maximum recommended dose) were 2800 ng/mL and 5960 ng*h/mL, respectively. Both C_max and AUC_0-inf appear to increase proportionally with increasing doses.