Phenylketonuria
Jul 29, 2025
Ascendis Pharma’s SKYTROFA Approved by FDA for Adult Growth Hormone Deficiency; Apellis Wins FDA Approval for EMPAVELI in C3G and IC-MPGN Treatment; FDA Approves PTC Therapeutics’ Sephience for Phenylketonuria; MAIA Biotechnology’s Ateganosine Gets FDA Fast Track for Non-Small Cell Lung Cancer; Entera Bio Gets FDA Nod on BMD as Primary Endpoint for Phase III Osteoporosis Study
Ascendis Pharma’s SKYTROFA Approved by FDA for Adult Growth Hormone Deficiency Ascendis Pharma A/S announced that the FDA has approved SKYTROFA (lonapegsomatropin-tcgd) for the treatment of adult growth hormone deficiency (GHD). Already approved for pediatric GHD since 2021, SKYTROFA is a once-weekly prodrug of ...
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Aug 06, 2024
Tecelra by Adaptimmune: First FDA-Approved Engineered Cell Therapy for Solid Tumors; GSK’s JEMPERLI Approved for Endometrial Cancer; MBX Biosciences Secures $63.5M in Series C for PEP™ Platform; FDA Stops Actinium’s Radiotherapy Blood Cancer Plans, Pushing Partnering Shift; Otsuka Acquires Jnana for Up to $1.1B, Enhancing Drug Discovery
Adaptimmune’s TECELRA Becomes First FDA-Approved Engineered Cell Therapy for Solid Tumors Adaptimmune Therapeutics announced that the FDA has approved (afamitresgene autoleucel), which will be marketed under the brand name TECELRA, for the treatment of unresectable or metastatic synovial sarcoma. This marks the ...
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Sep 21, 2021
Biogen’s AMD Biosimilar Gets Approval; SmartLabs Series B Funding; BARDA, Partner Therapeutics in Sepsis Space; Synlogic to Explore Sepsis Market Domain
Win-win for Biogen as its Biosimilar Gets Approval for AMD Samsung Bioepis and Biogen's biosimilar drug, Byooviz, received USFDA approval for the treatment of vision loss, Age-related macular degeneration and has become the first biosimilar to Lucentis in the AMD market. The Lucentis copycat had also manag...
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Sep 06, 2021
Which Pipeline Therapy is Expected to Capture the Eyeballs in the Phenylketonuria Market?
Phenylketonuria is an inherited disorder characterized by the absence or deficiency of an enzyme called phenylalanine hydroxylase (PAH), responsible for processing the amino acid phenylalanine. The condition affects one in 13,500 to 19,000 newborns in the United States, says NORD. DelveInsight’s Phenylketonuria epi...
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