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RSS FeedPosted by Michael Wonder on 28 Mar 2026
Egetis announces FDA acceptance and priority review of NDA for Emcitate (tiratricol) for MCT8 feficiency
27 March 2026 - Egetis Therapeutics today announced that the US FDA has accepted the filing of its new drug application ...
Posted by Michael Wonder on 16 Mar 2026
EMA proposes new paper on proof of concept studies for paediatric oncology drugs
13 March 2026 - The EMA has released a concept paper proposing a future reflection paper on the development of ...
Posted by Michael Wonder on 13 Mar 2026
Novartis Cosentyx receives FDA approval for paediatric patients aged 12+ with moderate to severe hidradenitis suppurativa
13 March 2026 - Novartis announced today that Cosentyx (secukinumab) received US FDA approval for treating paediatric patients 12 years ...
Posted by Michael Wonder on 28 Feb 2026
FDA approves Novo Nordisk's Sogroya as the first and only once weekly, long-acting growth hormone for three additional paediatric indications
27 February 2026 - Novo Nordisk today announced that the US FDA has approved three new indications for once weekly Sogroya ...
Posted by Michael Wonder on 28 Feb 2026
FDA approves once weekly Yuwivel (navepegritide) for children with achondroplasia aged 2 years and older
27 February 2026 - Rare paediatric disease priority review voucher granted in connection with approval. ...
Posted by Michael Wonder on 27 Feb 2026
Dupixent (dupilumab) recommended for EU approval to treat chronic spontaneous urticaria in young children with ongoing symptoms despite treatment
27 February 2026 - Regeneron Pharmaceuticals and Sanofi today announced that the EMA’s CHMP adopted a positive opinion recommending the approval ...
Posted by Michael Wonder on 06 Feb 2026
Teva Canada announces approval of expanded indication of Ajovy (fremanezumab solution for subcutaneous injection), the first anti-CGRP preventive treatment for paediatric episodic migraine
4 February 2026 - Teva Canada announced today that Health Canada has approved an expanded indication for Ajovy (fremanezumab solution for ...
Posted by Michael Wonder on 02 Feb 2026
Pharming Group receives complete response letter from US FDA for sNDA for Joenja (leniolisib) in children aged 4 to 11 years with APDS
1 February 2026 - Pharming today announced that the US FDA has issued a complete response letter to its supplemental ...
Posted by Michael Wonder on 12 Jan 2026
FDA approves first treatment for children with Menkes disease
12 January 2026 - The US FDA today approved the Zycubo (copper histidinate) injection as the first treatment for Menkes disease ...
Posted by Michael Wonder on 05 Jan 2026
Sanofi’s Tzield accepted for priority review in the US for young children with stage 2 type 1 diabetes
5 January 2026 - The US FDA has accepted for priority review the supplemental biologic license application for Tzield (teplizumab-mzwv) to ...
Posted by Michael Wonder on 20 Dec 2025
European Commission approves Tremfya (guselkumab) for the treatment of children with plaque psoriasis, marking the first paediatric indication for an IL-23 inhibitor
19 December 2025 - The decision is supported by the Phase 3 PROTOSTAR study, where guselkumab demonstrated higher levels of skin ...
Posted by Michael Wonder on 13 Dec 2025
BioCryst announces FDA approval of Orladeyo (berotralstat) oral pellets, first and only oral prophylactic treatment for patients with HAE aged 2 to <12 years
12 December 2025 - BioCryst Pharmaceuticals today announced that the US FDA has approved its new drug application for the ...
Posted by Michael Wonder on 08 Dec 2025
House passes bill that reauthorises the FDA’s paediatric priority voucher pathway
2 December 2025 - The US House of Representatives has passed a new piece of legislation that reauthorises the US ...
Posted by Michael Wonder on 02 Dec 2025
Solid Biosciences receives FDA rare paediatric disease designation for SGT-212 dual route of administration gene therapy for Friedreich’s ataxia
1 December 2025 - Solid Biosciences today announced that it received rare paediatric disease designation from the US FDA for SGT-212, ...
Posted by Michael Wonder on 01 Nov 2025
Johnson & Johnson submits application to US FDA for Stelara (ustekinumab) in the treatment of paediatric ulcerative colitis
31 October 2025 - Expanded Stelara indication seeks to treat children two years of age and older with moderately to severely ...