Tough Genes’ Post

We are pleased to announce that the FDA has granted Orphan Drug Designation and Rare Pediatric Disease Designation to our Ifetroban product candidate for the treatment of cardiomyopathy associated with Duchenne muscular dystrophy (DMD).    – Orphan Drug Designation is granted to products that address rare, or orphan, diseases. Such designation can result in reduced approval requirements, an expedited FDA review process, waiver of FDA filing fees and an extended exclusivity period after product approval.     – Rare Pediatric Disease Designation is given to products that address diseases that primarily affect children. This designation may result in a valuable priority review voucher from the FDA that can be used to accelerate approval of another product.    Securing these designations is a critical step forward for DMD, a devastating genetic disorder that affects young boys. These designations not only recognize the urgent need for effective treatments but also provide vital support to accelerate research and development.    Learn more: https://lnkd.in/eN3_Zi7b 

Glad to share our latest research focused on immunosuppression management in pediatric transplant recipients! In this study, we examined changes in immunosuppressive medications, particularly looking at the reductions, the rationale behind these adjustments, and their impact on alloimmunity. Despite reducing MMF dosages to nearly half of the intended levels due to side-effects, we observed a low rejection rate (4%) within the first year, and only a few cases of de novo donor-specific antibody (DSA) development during follow-up. This research contributes valuable insights into real-world challenges of balancing the immunosuppression for pediatric recipients. 📖 Check out the full study for more details on our approach and results! https://lnkd.in/d9qZAkhG

📢 FDA grants traditional approval to Lilly’s Retevmo (selpercatinib), for adult and pediatric patients 2 years of age and older with advanced or MTC with a RET mutation (Eli Lilly and Company) ➤ Selpercatinib previously had accelerated approval for this indication for patients 12 years of age and older in 2020. On May 29, 2024, the FDA granted accelerated approval for this indication to pediatric patients 2 years of age and older ➤ Efficacy was evaluated in LIBRETTO-531 (NCT04211337), a randomized, multicenter, open-label study in adults and adolescents with advanced or metastatic RET-mutant MTC ➤ Median PFS was not reached in the selpercatinib arm and 16.8 months (95% CI: 12.2, 25.1) in the cabozantinib/vandetanib arm (Hazard Ratio 0.280 [95% CI: 0.165, 0.475] p-value <0.0001) ➤ This application was granted priority review, breakthrough designation, and orphan drug designation #oncology #biotech #biotechnology #regulatoryaffairs #pharma #drugdevelopment #oncologytrials #oncologyresearch #cancer #oncology #regulatoryintelligence #regulatoryprecedent https://lnkd.in/e9bX7UVw

Race Oncology is pleased to share that the US Food and Drug Administration (US FDA) has extended Rare Paediatric Disease Designation (RPDD) to RC220 bisantrene for the treatment of paediatric acute myeloid leukemia (#AML). #RPDD is granted by the US FDA to new treatments of serious or life-threatening diseases that affect fewer than 200,000 people in the US, primarily those who are under 18 years old. The RPDD may qualify Race to receive a Priority Review Voucher (PRV) at the time of marketing approval or authorisation for RC220 in paediatric AML. The PRV can be redeemed for an accelerated review of RC220 by the US FDA, or sold to other parties, on average for more than US$100 million on the current open market. Race is currently in talks with world-renowned opinion leader in the treatment of AML, Associate Professor Himalee Sabnis, as well as an international paediatric oncology cooperative group, to further develop the company’s paediatric AML program. Dr Daniel Tillett, Race Chief Executive Officer, says: “US FDA RPDD is incredibly valuable as not only does it offer eligibility for the award of a PRV, but the ability to work with passionate clinicians and regulators to bring help to children and adolescents facing an enormously challenging disease with few effective treatment options.” More details, including an interview with our Executive Director Dr Peter Smith, are available here: https://lnkd.in/gTwX5c4r #paediatricAML #childhoodcancer #oncologytreatment #bisantrene

CF therapy ETD001 granted FDA’s rare pediatric disease designation ETD001, an investigational therapy aimed at improving mucus clearance in cystic fibrosis (CF) patients, has been granted the Rare Pediatric Disease Designation by the FDA! 🎉 This important milestone accelerates the development of treatments for children with life-threatening rare diseases, providing new hope for those with CF. ETD001 targets the epithelial sodium channel to improve lung function and mucus clearance, offering potential benefits to all CF patients, regardless of their mutation type. 🙌 A Phase 2a clinical trial is already underway to explore its safety and effectiveness. We're one step closer to better treatment options for CF! 💪 To read more the article: https://lnkd.in/gNR2JJec #CysticFibrosis #RareDisease #ETD001 #PediatricHealth #FDAApproval #MedicalInnovation #CFCommunity #EnterpriseTherapeutics #ClinicalTrials #HealthcareInnovation #CFAwareness #CFA

The United States Food and Drug Administration (US FDA) has extended the rare paediatric disease designation (RPDD) of the novel Race Oncology Ltd (ASX:RAC, OTC:RAONF) drug RC220 bisantrene for the treatment of paediatric subtypes of Acute Myeloid Leukaemia (AML). This designation was previously granted to RC110 bisantrene in 2018. RPDD is awarded for new treatments of serious or life-threatening diseases affecting fewer than 200,000 people in the US, primarily those under 18 years of age. Around 70% of rare diseases are exclusively paediatric in onset, with 95% having no approved treatments. The designation makes RC220 eligible for a priority review voucher (PRV) upon marketing approval, which can be transferred or sold. More at #Proactive #ProactiveInvestors #ASX #RAC #Oncology #Cancer #Biotech http://ow.ly/RXlG105xcvz

FDA has granted Rare Pediatric Disease Designation (RPDD) to a new proprietary investigational drug, 101-PCG-005, a type IA prodrug of dexamethasone targetsing CD206+ macrophages. The intended use is in systemic juvenile idiopathic arthritis (sJIA) or Still's disease .

As most drugs are developed for adult patients, often the doses for pediatric, geriatric and pregnant populations are unknown or are estimated based on body weight assuming PK to be similar. This is associated with a big uncertainty for efficacy and adverse events. PBPK models factor in physiology (system specific parameters) of these populations and integrate them with drug specific parameters to predict PK profiles. This is elegantly demonstrated for the bispecific drug Odronextamab for identification of doses in different categories of paediatric patients with relapsed/refractory B-cell NHL (B-NHL). Key factors considered were Clearance, Volume of distribution that were covariates in adult populations along with Target mediated drug disposition (TMDD) parameters. Odronextamab displays TMDD in adults. Similarity of target and tumor burden in adult and pediatric patients supported similar targeted-related nonlinear drug clearance. An excellent example of how MIDD is used in DDD. https://lnkd.in/gFNWUPAH

You are subscribed to Oncology (Cancer) / Hematologic Malignancies Approval. This information has recently been updated, and is now available. FDA grants accelerated approval to selpercatinib for pediatric patients two years and older with RET-altered metastatic thyroid cancer or solid tumors On May 29, 2024, the Food and Drug Administration granted accelerated approval to selpercatinib (Retevmo, Eli Lilly and Company) for pediatric patients two years of age and older with the following: advanced or metastatic medullary thyroid cancer (MTC) with a RET mutation, as detected by an FDA-approved test, who require systemic therapy; advanced or metastatic thyroid cancer with a RET gene fusion, as detected by an FDA-approved test, who require systemic therapy and who are radioactive iodine-refractory (if radioactive iodine is appropriate); and locally advanced or metastatic solid tumors with a RET gene fusion, as detected by an FDA-approved test, that have progressed on or following prior systemic treatment or who have no satisfactory alternative treatment options. #fda #approval #pediatric #oncology https://lnkd.in/gH5TdUVJ

👍 𝐅𝐃𝐀 𝐠𝐫𝐚𝐧𝐭𝐬 𝐚𝐜𝐜𝐞𝐥𝐞𝐫𝐚𝐭𝐞𝐝 𝐚𝐩𝐩𝐫𝐨𝐯𝐚𝐥 𝐭𝐨 𝐭𝐨𝐯𝐨𝐫𝐚𝐟𝐞𝐧𝐢𝐛 𝐩𝐚𝐭𝐢𝐞𝐧𝐭𝐬 6 𝐦𝐨𝐧𝐭𝐡𝐬 𝐨𝐟 𝐚𝐠𝐞 𝐚𝐧𝐝 𝐨𝐥𝐝𝐞𝐫 𝐰𝐢𝐭𝐡 𝐫𝐞𝐥𝐚𝐩𝐬𝐞𝐝 𝐨𝐫 𝐫𝐞𝐟𝐫𝐚𝐜𝐭𝐨𝐫𝐲 𝐁𝐑𝐀𝐅-𝐚𝐥𝐭𝐞𝐫𝐞𝐝 𝐩𝐞𝐝𝐢𝐚𝐭𝐫𝐢𝐜 𝐥𝐨𝐰-𝐠𝐫𝐚𝐝𝐞 𝐠𝐥𝐢𝐨𝐦𝐚. This represents the first FDA approval of a systemic therapy for the treatment of pediatric patients with relapsed or refractory pediatric low-grade glioma (LGG) harboring a BRAF fusion or rearrangement, or BRAF V600 mutation. This application was granted priority review, breakthrough designation and orphan drug designation. 👶 Pediatric patients often have limited treatment options, so expedited drug development can make a huge difference in their lives. By receiving priority review, these pediatric patients can access potentially life-saving treatments much faster than before: 𝑩𝒆𝒄𝒂𝒖𝒔𝒆 𝑪𝒉𝒊𝒍𝒅𝒓𝒆𝒏 𝑪𝒂𝒏'𝒕 𝑾𝒂𝒊𝒕! https://lnkd.in/eQPak_N9 #childrencantwait #pediatricclinicaltrials