Proactive’s Post

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

Spinal Muscular Atrophy is a tough disease.  So it’s good that Scholar Rock’s apitegromab 💉 Just succeeded in a Phase III clinical trial! 📊 “Scholar Rock’s stock has soared off the back of positive Phase III results from its spinal muscular atrophy (SMA) candidate. The Phase III SAPPHIRE trial (NCT05156320) evaluating Scholar Rock’s lead pipeline candidate apitegromab, met its primary endpoint of statistically significant and clinically meaningful improvement in motor function, as measured by the Hammersmith Functional Motor Scale Expanded (HFMSE). The company’s stock price rocketed by 362% after the news dropped, from a 4 October close of $7.42 to a close of $34.28 (7 October). Apitegromab is a monoclonal antibody that inhibits myostatin activation by selectively binding the pro- and latent forms of myostatin in the skeletal muscle. It is the first muscle-targeted treatment candidate to demonstrate clinical proof-of-concept in SMA. Kenneth Hobby, President of Cure SMA, said: “It’s a great day for people living with SMA and their families. . Declining motor function and hopes for reversing losses associated with muscle weakness are significant unmet needs, impacting activities of daily living, from breathing, eating, self-care, to working and social interactions. We need an approved therapy.” In the main population, aged 2-12, the mean difference in change from baseline in HFMSE was 1.8 points for all patients receiving apitegromab, compared to placebo, with improvement measured as early as eight weeks. Patients receiving 20mg/kg of apitegromab showed a 1.4-point difference while the 10mg/kg cohort showed an improvement of 2.2 points. The exploratory population, aged 13-21, also favoured apitegromab compared to placebo in motor function improvement. There was more than a three-point improvement in HFMSE in 30% of #apitegromab patients versus 12.5% of patients on placebo. Treatment was well tolerated, and the safety profile was consistent with previous studies. Following trial completion, 98% of SAPPHIRE patients enrolled in the ongoing ONYX open-label expansion study. FDA granted Fast Track, Orphan Drug, and Rare Paediatric Disease designations to apitegromab while European Medicines Agency (EMA) has granted Priority Medicines (PRIME) and Orphan Medicinal Product designations. Analyses of the full Phase III SAPPHIRE data are ongoing, with a detailed presentation to take place at a 2025 medical conference. Scholar Rock is also investigating apitegromab alongside a glucagon-like peptide-1 receptor agonist (GLP-1ra) in obese patients in a Phase II. SMA is a rare disorder that results in loss of motor neurons and progressive muscle wasting. It is usually diagnosed in infancy or early childhood. Current treatments include #Spinraza (nusinersen), #Evrysdi (risdiplam) and #Zolgensma (onasemnogene abeparvovec)." Thanks to Abigail Beaney for Clinical Trials Arena - article link in the comments! #clinicaltrials #SMA #spinalmuscularatrophy

Innorna Announces U.S. FDA Rare Pediatric Disease Designation Granted to IN022 for the Treatment of Homocystinuria HONG KONG, SHENZHEN, NANJING, CHINA, July 08, 2024--Innorna, a clinical-stage biotech company pioneering its proprietary lipid nanoparticle (LNP) technology to develop novel RNA therapeutics, today announced that the U.S. Food and Drug Administration (FDA) has granted Rare Pediatric Disease Designation (RPDD) to its investigational therapy IN022 for the treatment of classic homocystinuria (HCU), a serious or life-threatening genetic disease. This RPDD will greatly facilitate IN022 clinical development and bring this potential therapy to patients as quickly as possible. The RPDD also means that FDA may award a Priority Review Voucher upon the approval of the IN022 product. About HCU and IN022 HCU is a rare autosomal recessive inherited disorder of sulfur amino acid metabolism primarily caused by deficiency of cystathionine beta synthase (CBS). HCU patients have a wide spectrum of age (older than three years) on presentation, with multisystemic clinical complications, particularly skeletal and connective tissue defects, osteoporosis, dislocated optic lenses, learning difficulties, and developmental delay or thromboembolism. Currently, there is no approved therapeutic drug for this disease. IN022 is designed to address the root cause of HCU resulting from deficiency in CBS. It is expected to restore the function of CBS enzyme, thereby normalizing homocysteine metabolism and potentially ameliorating symptoms in HCU patients.

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 

Takeda’s Takhzyro (lanadelumab) has been approved by the Medicines and Healthcare products Regulatory Agency (MHRA) to prevent recurrent attacks of hereditary angioedema (HAE) in patients aged two to less than 12 years. The regulator’s decision expands Takhzyro’s previous paediatric indication in patients aged 12 years and older and makes the drug the first long-term prophylactic treatment of HAE available in the UK for children aged two to under six years. Estimated to affect one in every 50,000 people, HAE is a rare genetic disorder characterised by recurring oedema (swelling) attacks in various parts of the body, including the abdomen, face, hands and throat. The swelling can be debilitating and painful, and attacks that obstruct the airways can cause asphyxiation and are potentially life-threatening. “HAE, like so many other rare diseases, is highly complex, and patients, their families and caregivers often undergo years of strain trying to understand their disease, get a definitive diagnosis and gain access to the medicines they need,” Takeda said in a statement. Takhzyro, which can be administered subcutaneously by patients or their caregivers following appropriate training, is a fully human monoclonal antibody that helps to prevent the swelling and related symptoms of HAE by binding to and blocking the action of an enzyme in the blood called kallikrein. The MHRA’s latest approval of the therapy is supported by results from the late-stage SPRING study in combination with extrapolation of data from the pivotal adult and adolescent HELP study DX-2930-037 and by data supporting the new 150mg pre-filled syringe formulation. Commenting on the decision, Angela Metcalfe, chief executive officer of HAE UK, said: “The announcement that children under 12 now have access to this treatment option will be looked back on as a huge milestone for the HAE community. Read more: https://lnkd.in/eR3WEB3r Stay in touch with all the leading stories, events and opportunities by subscribing to our LinkedIn Newsletter:https://bit.ly/3RbdKtc or joining some of the largest groups most relevant to you: https://bit.ly/4caKquL (A-Z list)

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

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

Ceres Brain Therapeutics SAS is proud to announce that the FDA has granted rare Pediatric Designation from our leading asset, CBT101, for the treatment of Creatine Transporter Deficiency Syndrome (CTD). This opens the door to potentially receiving a Priority Review Voucher, a key step in accelerating the development of our innovative treatments. Stay tuned for more updates! We remind you that CTD is an X-linked gene disease resulting in severe intellectual deficiency, autistic syndrome and seizure due to lack of creatine in brain neurons. CBT101, is a prodrug of creatine that delivers creatine to brain neurons by following the nose-to-brain pathway after nasal administration. CBT101 demonstrated particularly strong efficacy in two different mice models of CTD, increasing brain plasticity, improving brain metabolism and restoring cognition. https://lnkd.in/e4ka-7uY

📢 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

Tough Genes, along with over 200 other organizations, proudly signed a joint letter calling Congress to approve a long-term extension of the Priority Review Voucher program through the Creating Hope Reauthorization Act. What does this mean and why is this important to us? Developing therapeutic drug treatments for rare pediatric disorders is challenging. Faced with small patient populations, delays in diagnosis and difficulties conducting clinical trials for children, the average time the FDA takes to approve a drug for use is over 15 years. According to the EveryLife Foundation, a cowriter for the letter, “49 Rare Pediatric PRVs have been issued since 2012 for innovative treatments in 40 diseases like spinal muscular atrophy, Duchenne muscular dystrophy, and progeria syndrome. 7 out of 7 drug developers interviewed by the GAO reported that PRVs were a factor in drug development decisions.” What this means is, thanks to PRVs, lifesaving, or life enhancing therapeutic drugs have been made available for 40 diseases and countless patients. Over the past decade, the Rare Pediatric Disease (RPD) Priority Review Voucher (PRV) program has been an invaluable tool, expediting new treatments for children with rare conditions and genetic disorders just like IRF2BPL-Related Disorder. We thank the EveryLife Foundation and the National Organization for Rare Disorders for collaborating, writing and distributing this letter.