DDC dopa decarboxylase

For eladocagene exuparvovec, foscarbidopa, benserazide: what EMA and FDA publish about paediatric plans, orphan status and pediatric approvals, and how many registered studies take children. Each line is a yes, no or not published fact from the record linked under it; none of it is a forecast.

• FDA pediatric target list: no · DDC is not on the list

  Rule: Target is on FDA's Pediatric Molecular Target List (relevant targets). Source: FDA Pediatric Molecular Target List (Listed as February 2026 on the FDA Pediatric Oncology page; the file served is 9.7.22_the_relevant_molecular_target_list.xlsx, whose Summary of Changes sheet is headed 'SEPTEMBER 2022 UPDATE' (footnote date 2022-09-01)), 2026-11-11
• EU paediatric investigation plan: yes · EMEA-002435-PIP01-18-M03 (Eladocagene exuparvovec, Treatment of aromatic L-amino acid decarboxylase deficiency): PM: decision on the application for modification of an agreed PIP, 10 Mar 2023; compliance positive 26 May 2023

  Rule: An EMA PIP decision (agreement, modification or waiver) lists the drug as its active substance, exact name. Source: EMA EMEA-002435-PIP01-18-M03, 2026-11-11
• EU orphan designation: yes · EU/3/16/1786 positive 18 Nov 2016: Treatment of aromatic L-amino acid decarboxylase deficiency; EU/3/18/2125 positive 11 Jan 2019: The treatment of sickle cell disease; EU/3/14/1402 positive 16 Dec 2014: Treatment of beta thalassaemia intermedia and major

  Rule: An EU orphan designation lists the drug as its active substance (exact name) or belongs to a medicine that does; withdrawn and expired designations are listed with their status. Source: EMA data download (dated snapshot), 2026-11-11
• Authorised in the EU: yes · Upstaza: authorised since 18 Jul 2022, orphan medicine, exceptional circumstances

  Rule: An EMA medicine record lists the drug as its active substance or INN, exact name; national-only authorisations are not in this file. Source: EMA EPAR Upstaza, 2026-11-11
• FDA orphan designation: not published · FDA's orphan designation database turns away automated requests, so this app does not read it; search it by hand at the link

  Rule: Would be: an FDA orphan designation names the drug. Source: FDA orphan designations search
• FDA pediatric oncology approval: no · FDA's table lists no pediatric approval of eladocagene exuparvovec, foscarbidopa, benserazide

  Rule: FDA's Pediatric Oncology Drug Approvals table names the drug. Source: FDA Pediatric Oncology Drug Approvals (content current as of 2026-03-03), 2026-11-11
• Registered studies open to children: yes · 2 interventional studies of eladocagene exuparvovec, foscarbidopa, benserazide include patients under 18; 1 is recruiting, not yet recruiting or active

  Rule: The registry's age group includes Child (birth to 17 years), which also counts studies that set no minimum age. Source: ClinicalTrials.gov search, 2026-11-11

Rare pediatric disease voucher. FDA, content current as of 29 Apr 2026: “As of February 3, 2026, enactment of the Consolidated Appropriations Act, 2026, the rare pediatric disease PRV program will sunset after September 30, 2029. FDA may not award any PRVs under this program after September 30, 2029.” Voucher sale prices are not shown here; they appear only as company-reported news. FDA: Rare Pediatric Disease Designation and Priority Review Voucher Programs

Each drug, stage and indication links to its Open Targets record, where the trials and approvals behind it are listed. Salt forms (for example a mesylate) are listed as Open Targets records them, as separate entries.