ProfoundBio’s Post

"On Friday, February 23, 2024, FDA announced its final decision to withdraw the approval of Pepaxto (melphalan flufenamide), which was approved in February 2021 in combination with dexamethasone for the treatment of adult patients with relapsed or refractory multiple myeloma (RRMM) who have received at least four prior lines of therapy and whose disease is refractory to at least one proteasome inhibitor, one immunomodulatory agent, and one CD38-directed monoclonal antibody. This is the third withdrawal of an accelerated approval FDA has performed, following Avastin in 2011 and Makena in 2023. Notably, this decision marks the first use of the new expedited procedures for withdrawal of an accelerated approval that were enacted in the Food and Drug Omnibus Reform Act of 2022 (FDORA). Prior to FDORA’s enactment, the statute did not provide details on the withdrawal procedures other than offering an opportunity for an informal hearing." #FDA #AcceleratedApproval #DrugDevelopment https://lnkd.in/esJ-dZGF

It is important to have a proactive approach when it comes to minimizing obstacles that are associated with drug development for rare diseases. This is to ensure access to more therapies that address rare diseases. In support of these efforts while also encouraging innovation the US FDA’s CDER collaborated to review and evaluate educational opportunities across various topics in rare disease drug development. This blog covers the methodology and key findings that came out from the Learning and Education to Advance and Empower Rare Disease Drug Developers, or LEADER 3D https://lnkd.in/gxXkmaEc Meet us at the BIO International Convention in San Diego to discuss your regulatory requirements for rare disease drug approval. Schedule a meeting with us https://lnkd.in/gJt7Vxwv Write to DDReg on info@ddregpharma.com or submit your query here https://lnkd.in/gxXkmaEc #raredisease #pharma #pharmaceuticalmanufacturing #innovation #drugdevelopment #empowering #USFDA #BIO2024

At the World Orphan Drug Congress, Michael Fusakio and Samantha Z. held a fireside chat, ‘Rare but Not Forgotten: Challenges and Opportunities for Rare Disease Drugs,’ and talked through the challenges and implications in drug development for #rarediseases. They shared their own stories so that others could walk away with practical suggestions because developing therapies for rare diseases certainly has its own unique set of development and regulatory hurdles. But opportunities await! Here's a look into their Q&A. For questions about your clinical development plan and regulatory strategy, we’re ready when you are. https://hubs.la/Q02vYNF50 #WODC #RareDisease #RareDiseaseAwareness #ClinicalDevelopment #ClinicalTrials

Amgen has secured the FDA’s green light for the first interchangeable biosimilar to AstraZeneca’s Soliris to treat two rare diseases. The biosimilar will carry the brand name Bkemv. Like its branded reference product, Bkemv is indicated for the treatment of paroxysmal nocturnal hemoglobinuria and atypical hemolytic uremic syndrome. It also carries a boxed warning for serious meningococcal infections, which according to its label can be “serious and life-threatening.” Because of this risk, Bkemv is only available through a restricted Risk Evaluation and Mitigation Strategies program. Bkemv was also granted the FDA’s interchangeability designation, which allows it to be used in place of the branded reference product without needing to change the prescription. Depending on state laws, this substitution may take place at the pharmacy. #biotech #biopharma #Soliris #RareDiseases #FDA #Approval

This review of #realworldevidence focused on #FDA approved products for #raredisease is worth a read - my thoughts: 1) Most #realworlddata supported natural history use cases (70% - based on 243 applications screened and 20 applications which went through detailed review) 2) "Despite the FDA’s willingness to accept RWE in drug approval assessment, there remains a lack of knowledge among stakeholders regarding the agency’s acceptance of RWE for certain orphan indication approvals" 3) Table 2: "Characteristics of real-world data (RWD) used in FDA review packages" breaks down each approval with key details (eg, indication, study design, purpose, sample size) 4) Only 65% of the RWD studies had documentation stating that an a priori protocol was in place --- this is a growth area for those planning to include RWD/E in regulatory submissions 5) Table 3: "FDA feedback on submissions and RWD reported in FDA labels" breaks down the primary clinical studies and FDA's written feedback as well as a summary of RWD's inclusion in the label - FDA's feedback on RWD was favorable for about half of these submissions (45%) 6) Even when FDA had criticisms, RWD was often included in the approved label alongside the primary clinical study(ies) citation: Vaghela, S., Tanni, K.A., Banerjee, G. et al. A systematic review of real-world evidence (RWE) supportive of new drug and biologic license application approvals in rare diseases. Orphanet J Rare Dis 19, 117 (2024). https://lnkd.in/eNZ2cGxk

GSK and Aiolos Bio have entered into agreement under which GSK will acquire Aiolos, for a $1B upfront and up to $400M in certain success-based regulatory milestone payments. AIO-001 is a phase-2 ready anti-TSLP with q6m dosing I'd say $1B upfront is a huge risk given the clinical development hurdles it has to pass Is q6m dosing really going to be a gaming changing USP by the time it approved ? By then Teze will be well entrenched with likely indications also for CRSwNP and T2 high /lowCOPD Not to mention Dupilumab in T2 high asthma/COPD/CRSwNP/AD/EoE -ie the bar will set high for any new entity in T2 high disease Bear in mind the anti-TSLP space will likely get crowded: UPSTREAM BIO have their anti-TSLP UPB-101 q12-24 wk dosing ready to roll with Phase 2 BIOSION BSI-045B anti-TSLP also set for phase 2 asthma and AD (where Teze has already failed)

89bio wins EMA priority status for #MASH candidate #pegozafermin: The European Medicine’s Agency (EMA) has granted priority medicines (PRIME) status to 89bio’s pegozafermin, a drug being evaluated to treat patients with non-cirrhotic metabolic dysfunction-associated steatohepatitis (MASH) with advanced forms of fibrosis. The EMA’s PRIME scheme is designed to expedite the development of medicines that meet an unmet need, such as rare diseases. The agency upgraded the scheme last year to enhance scientific advice and regulatory package preparation for sponsors of the scheme. 89bio’s pegozafermin was granted PRIME status based on positive data from the company’s Phase IIb ELIVEN trial (NCT04929483), according to a 27 March press release. The randomised trial saw three doses of the drug administered either once weekly or biweekly in 192 patients with non-cirrhotic MASH with fibrosis (F2-F3) and MASH with compensated cirrhosis (F4). https://lnkd.in/gAXvNH2P #RegulatoryCompliance #Regulatorybody #RegulatoryIntelligence

Island Pharmaceuticals is pleased to share that the US Food and Drug Administration (FDA) has cleared the proposed protocol amendments for its Phase 2a/b clinical trial for ISLA-101, Island’s lead drug candidate, in dengue fever.    Using a human challenge study model, the Phase 2a/b trial will explore both prophylactic (prevention) and therapeutic arm split across two cohorts, and has been renamed PROTECT (PROphylactic and TrEatment Challenge Trial). As part of the consultation process on the protocol, the FDA requested that Island wait to start infecting subjects until the end of mosquito season, which occurs on October 1.   Following FDA advice, Island plans to start dosing the Phase 2a (prophylactic) cohort in late September and start infecting around October 1, expecting full readout well before the end of the year. Phase 2b will begin being dosed in January 2025. ISLA-101 will be administered at a single dose level, twice daily across multiple days.   Island has received ethics approval from the Institutional Review Board (IRB) at SUNY Update New York; steps seeking IRB approval from the US Army are currently underway. Island is pleased to advise that the cost to Island for the full study is around US $1.08m, substantially lower than prior estimates in view of grant support as announced in May this year.   Island CEO and Managing Director Dr David Foster said: “We have worked hard to bolster our internal capabilities and as a result have not needed to engage a CRO for this study. This strategy not only has streamlined efforts on protocol revisions, FDA communications and trial execution, but has substantially reduced costs enabling us to complete the Phase 2a, prophylactic cohort of the PROTECT study with existing funds.”   For more details on the PROTECT Phase 2a/b clinical trial, read the full announcement via this link: https://lnkd.in/gFscYhqN #mosquitobornedisease #denguefever #antiviraltherapeutics

Noxopharm Limited (ASX:NOX, OTC:NOXOF) is preparing to launch a first-in-human Phase 1 trial assessing SOF-SKN™ drug candidate and Sofra™ platform for #AutoimmuneDiseases in its HERACLES or ‘Harnessing Endogenous Regulators Against CLE Study’. NOX sees the Australia-based trial as the first step in leveraging the Sofra™ platform’s potential to tackle the autoimmune disease market in areas such as #RheumatoidArthritis. Holding the trials within Australia will offer access to R&D tax incentives from the Federal Government and Australian expertise in #lupus research and early-phase clinical trials. The trial is planned to start in early 2025 and is designed to provide proof of concept for the skin disease that is caused by cutaneous lupus erythematosus (CLE). More at #Proactive #ProactiveInvestors #Biotech #Biopharmaceutcials #ASX #NOX http://ow.ly/4ENI105FPZa

We have just over a month to go until the first international drug repurposing conference, and we can’t wait to see you there! 📅 6th - 7th March 2024 📍 Barcelona Not got your ticket yet and need more convincing? 🤔 The conference, in collaboration with REMEDi4ALL and MeRIT, will explore a variety of repurposing projects with a dedicated rare disease pathway. One of the sessions is: 💊 Why care about repurposing for rare diseases? Advancing research in rare diseases is a challenging task. The high costs of drug development, limited knowledge about these conditions, and small patient populations, make researching rare disease treatments less attractive than common conditions. Where traditional drug development often fails, drug repurposing offers a promising path to success for rare diseases. This session will delve into the potential of repurposing in rare diseases, benefitting stakeholders from patient groups to the pharmaceutical industry. Early bird tickets are available until 31st January! 👇 https://ow.ly/VWVa50Qu4r4