dm+d

Unassigned

New Medicines

Nulibry (US) Metabolic disease molybdenum cofactor deficiency (MoCD) Type A

Information

Nulibry (US)
New molecular entity
BridgeBio Pharma
BridgeBio Pharma

Development and Regulatory status

Phase III Clinical Trials
Phase III Clinical Trials
Approved (Licensed)
Yes
Yes
Mar 21Approved in US [17]
Sep 20The FDA has accepted a New Drug Application for fosdenopterin a substrate replacement therapy, for the treatment of patients with molybdenum cofactor deficiency (MoCD) Type A [15]
Jul 20Company annual report indicates a rolling NDA filing initiated late 2019. No indication of plans for EMA filing [14].
Sep 19BridgeBio Pharma announces intention to submit NDA to the US FDA for Molybdenum cofactor deficiency, in 2020 [12]
Jan 18Alexion is ´de-prioritising´ ALXN-1101 as part of redefining its R&D strategy and will seek to licence it out to another company [10]. Timescale for licensing is therefore uncertain.
Jun 17BridgeBio Pharma enters into an agreement with Alexion to acquire cyclic pyranopterin monophosphate. BridgeBio has committed sufficient resources to Origin Biosciences (their new subsidiary) to enable clinical development, regulatory approval and to support commercialisation of cyclic pyranopterin monophosphate [11].
Feb 16PII/III study NCT02629393 initiated [7]
Jan 16 US orphan designation awarded [6]. Sep 10: EU orphan designation awarded [5].
Jan 16still listed as ´Early clinical development´ in Alexion´s pipeline [4].
Oct 13FDA awards breakthrough designation [1].

Category

A synthetic version of cPMP
MoCD is a severe and life-threatening, ultra-rare, genetic metabolic disorder: it results from a lack of molybdenum co-factor, for which cyclic pyranopterin monophosphate (cPMP) is a precursor. It causes catastrophic and irreversible neurologic damage within the first weeks of life and is usually fatal in early childhood. Estimated incidence is between 1 in 100,000 and 1 in 200,000, although this may be an underestimate [13].
Metabolic disease molybdenum cofactor deficiency (MoCD) Type A
Intravenous

Trial or other data

Nov 20Has been added to the 2020/21 National Tariff Payment System list of high cost drugs, using its previous name cyclic pyranopterin monophosphate [16].
Jan 17NCT02629393 still listed as recruiting [8].
Feb 16 a PII/IIII trial initiated (NCT02629393) with recruitment in the US, EU, and other countries for an estimated study population of 5 subjects; study will be open label and the primary outcome is that the patient is alive and able to sit upright independently for at least 30 seconds ans estimated primary outcome completion date is September 2018 [8].
Sep 15A cohort study of 16 newborn babies, published in the Lancet, reported that cPMP substitution in type A disease rapidly improved under treatment and convulsions were either completely suppressed or substantially reduced. Three patients treated early remained seizure free and showed near-normal long-term development. There was no biochemical or clinical response in patients with type B disease [3].
Apr 14Open-label PII trial initiated to assess the safety and efficacy of escalating daily doses in paediatric patients with MoCD type A currently receiving recombinant Escherichia coli-derived cPMP (NCT02047461). The trial will enrol 4 patients in the US, the UK, Australia and the Netherlands [2].