{{Short description|Thymus tissue medical therapy}} {{Use American English|date=October 2021}} {{Use dmy dates|date=October 2021}} {{Infobox drug | drug_name = | INN = | type = <!-- empty --> | image = | width = | alt = | caption = <!-- Clinical data --> | pronounce = | tradename = Rethymic | Drugs.com = | MedlinePlus = | licence_EU = <!-- EMA uses INN (or special INN_EMA) --> | DailyMedID = Allogeneic_processed_thymus_tissue | licence_US = <!-- FDA may use generic or brand name (generic name preferred) --> | pregnancy_AU = <!-- A / B1 / B2 / B3 / C / D / X --> | pregnancy_AU_comment = | pregnancy_category = | routes_of_administration = | class = | ATCvet = | ATC_prefix = None | ATC_suffix = | ATC_supplemental = <!-- Legal status --> | legal_AU = <!-- S2, S3, S4, S5, S6, S7, S8, S9 or Unscheduled --> | legal_AU_comment = | legal_BR = <!-- OTC, A1, A2, A3, B1, B2, C1, C2, C3, C4, C5, D1, D2, E, F --> | legal_BR_comment = | legal_CA = <!-- OTC, Rx-only, Schedule I, II, III, IV, V, VI, VII, VIII --> | legal_CA_comment = | legal_DE = <!-- Anlage I, II, III or Unscheduled --> | legal_DE_comment = | legal_NZ = <!-- Class A, B, C --> | legal_NZ_comment = | legal_UK = <!-- GSL, P, POM, CD, CD Lic, CD POM, CD No Reg POM, CD (Benz) POM, CD (Anab) POM or CD Inv POM / Class A, B, C --> | legal_UK_comment = | legal_US = Rx-only | legal_US_comment = <ref name="Rethymic FDA label">{{cite web | title=Rethymic- allogenic thymocyte-depleted thymus tissue-agdc implant | website=DailyMed | url=https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=e0022c28-8cda-4f1e-bcf1-1f440d37ec4a | access-date=20 December 2021}}</ref> | legal_EU = | legal_EU_comment = | legal_UN = <!-- N I, II, III, IV / P I, II, III, IV --> | legal_UN_comment = | legal_status = <!-- For countries not listed above -->
<!-- Pharmacokinetic data -->| bioavailability = | protein_bound = | metabolism = | metabolites = | onset = | elimination_half-life = | duration_of_action = | excretion = <!-- Identifiers --> | CAS_number_Ref = | CAS_number = | CAS_supplemental = | PubChem = | IUPHAR_ligand = | DrugBank_Ref = | DrugBank = | ChemSpiderID_Ref = | ChemSpiderID = | UNII_Ref = | UNII = | KEGG_Ref = | KEGG = | KEGG2_Ref = | KEGG2 = | ChEBI_Ref = | ChEBI = | ChEMBL_Ref = | ChEMBL = | NIAID_ChemDB = | PDB_ligand = | synonyms = RVT-802,<ref>{{cite press release | title=Enzyvant Resubmits Biologics Licensing Application (BLA) to FDA for RVT-802 for Pediatric Congenital Athymia | publisher=Enzyvant | via=GlobeNewswire | date=27 April 2021 | url=https://www.globenewswire.com/en/news-release/2021/04/27/2217439/0/en/ENZYVANT-Resubmits-Biologics-Licensing-Application-BLA-to-FDA-for-RVT-802-for-Pediatric-Congenital-Athymia.html | access-date=8 October 2021}}</ref> allogeneic processed thymus tissue-agdc
<!-- Chemical and physical data -->| IUPAC_name = | chemical_formula_ref = | chemical_formula = | C = | H = | Ag = | Al = | As = | Au = | B = | Bi = | Br = | Ca = | Cl = | Co = | F = | Fe = | Gd = | I = | K = | Li = | Mg = | Mn = | N = | Na = | O = | P = | Pt = | S = | Sb = | Se = | Sr = | Tc = | Zn = | charge = | molecular_weight = | molecular_weight_comment = | SMILES = | StdInChI = | StdInChI_comment = | StdInChIKey = | density = | density_notes = | melting_point = | melting_high = | melting_notes = | boiling_point = | boiling_notes = | solubility = | sol_units = | specific_rotation = }}
'''Allogeneic processed thymus tissue''', sold under the brand name '''Rethymic''', is a thymus tissue therapy used for the treatment of children with congenital athymia.<ref name="FDA PR">{{cite press release | title=FDA Approves Innovative Treatment for Pediatric Patients with Congenital Athymia | website=U.S. Food and Drug Administration (FDA) | date=8 October 2021 | url=https://www.fda.gov/news-events/press-announcements/fda-approves-innovative-treatment-pediatric-patients-congenital-athymia | archive-url=https://web.archive.org/web/20211008225527/https://www.fda.gov/news-events/press-announcements/fda-approves-innovative-treatment-pediatric-patients-congenital-athymia | archive-date=8 October 2021 | access-date=8 October 2021}} {{PD-notice}}</ref> It is administered via a one-time surgical procedure where the tissue slices are implanted into the quadriceps muscle.<ref name="Rethymic FDA label" /> It takes six months or longer to reconstitute the immune function in treated people.<ref name="FDA PR" />
The most common adverse reactions include high blood pressure, cytokine release syndrome, low blood magnesium levels, rash, low platelets, and graft versus host disease.<ref name="FDA PR" />
It was approved for medical use in the United States in October 2021.<ref name="FDA PR" /><ref>{{cite press release | title=Enzyvant Receives FDA Approval for Rethymic (allogeneic processed thymus tissue-agdc), a One-Time Regenerative Tissue-Based Therapy for Pediatric Congenital Athymia | publisher=Enzyvant | via=GlobeNewswire | date=8 October 2021 | url=https://www.globenewswire.com/en/news-release/2021/10/09/2311432/0/en/Enzyvant-Receives-FDA-Approval-for-RETHYMIC-allogeneic-processed-thymus-tissue-agdc-a-One-Time-Regenerative-Tissue-Based-Therapy-for-Pediatric-Congenital-Athymia.html | access-date=8 October 2021}}</ref><ref name="Enzyvant PR">{{cite press release | title=Enzyvant Receives FDA Approval for Rethymic (allogeneic processed thymus tissue-agdc), a One-Time Regenerative Tissue-Based Therapy for Pediatric Congenital Athymia | website=Enzyvant | date=8 October 2021 | url=https://enzyvant.com/enzyvant-receives-fda-approval-for-rethymic-allogeneic-processed-thymus-tissue-agdc-a-one-time-regenerative-tissue-based-therapy-for-pediatric-congenital-athymia/ | access-date=8 October 2021}}</ref> Allogeneic processed thymus tissue is the first thymus tissue product approved by the U.S. Food and Drug Administration (FDA).<ref name="FDA PR" />
Allogeneic processed thymus tissue is composed of human allogeneic (donor-derived) thymus tissue that is processed and cultured, and then implanted into people to help reconstitute immunity (improve immune function) in people who are athymic.<ref name="FDA PR" /><ref name="Kreins_2021">{{cite journal | vauthors = Kreins AY, Bonfanti P, Davies EG | title = Current and Future Therapeutic Approaches for Thymic Stromal Cell Defects | journal = Frontiers in Immunology | volume = 12 | issue = | article-number = 655354 | date = 2021 | pmid = 33815417 | pmc = 8012524 | doi = 10.3389/fimmu.2021.655354 | doi-access = free }}</ref> Dosing is patient customized, determined by the surface area of the allogeneic processed thymus tissue slices and the body surface area of the patient.<ref name="FDA PR" />
== Medical uses == Allogeneic processed thymus tissue is indicated for immune reconstitution in children with congenital athymia.<ref name="Rethymic FDA label" /><ref name="Enzyvant PR" />
== History == The safety and efficacy of allogeneic processed thymus tissue were established in clinical studies that included 105 participants, with ages from one month to 16 years, who each received a single administration of allogeneic processed thymus tissue, from 1993 to 2020.<ref name="FDA PR" /> Allogeneic processed thymus tissue improved survival of people with congenital athymia, and most people treated with this product survived at least two years.<ref name="FDA PR" />
The U.S. Food and Drug Administration (FDA) granted the application for allogeneic processed thymus tissue a rare pediatric disease voucher and granted approval of Rethymic to Enzyvant Therapeutics, Inc.<ref name="FDA PR" />
== References == {{reflist}}
{{HHS content|url=http://www.fda.gov}}
== Further reading == * {{cite journal |vauthors=Markert ML, Gupton SE, McCarthy EA |title=Experience with cultured thymus tissue in 105 children |journal=J Allergy Clin Immunol |volume= 149|issue= 2|pages= 747–757|date=August 2021 |pmid=34362576 |doi=10.1016/j.jaci.2021.06.028 | doi-access=free | title-link=doi |pmc=8810898 }}
Category:Congenital disorders Category:Immunology Category:Medical treatments Category:Orphan drugs Category:Thymus