Septa Pharma’s Post

#raredisease #drugdevelopment is really hard and inherently risky - and it can be tremendously rewarding! When else do you have a chance to bring the first treatment - and a glimmer of hope - to a community affected by really serious conditions, with incredible unmet medical need, and an almost unprecedented resolve to change the world. Rare disease drug development is a big priority for FDA. Great primer and summary of many of the efforts #CDER is undertaking to help spur rare disease drug development published this week: https://lnkd.in/eZRs3Dsv

#RareDiseaseWeek2024 I am so honored to be advocating in DC this week, with 800 peers from all 50 states! We are asking legislators to 1) create an Interagency Collaboration Committee for Rare Diseases, & 2) to fund additional Rare Disease research for early diagnosis, treatment, & education for medical professionals. Fact 1: There are more than 10,000 known rare diseases and only a few hundred have safe, effective treatments...many still have NO treatments. Only 5% have FDA approved pharmaceutical drugs. #RareDC2024 #BeTheChange #RDLA Rare Disease Day EveryLife Foundation for Rare Diseases National Organization for Rare Disorders Disability:IN The Ehlers-Danlos Society Photo of several hundred people on the steps of the US Capitol building with the caption Rare Disease Week on Capitol Hill - Rare Disease Legislative Advocates, presented by Amgen

Another important step completed - with some valuable experience of the new(ish) CTIS clinical trial approval system gained along the way!

Download our report about #RareDisease and Orphan drugs.

Rare Disease Day unites the global #raredisease community to address challenges and promote equity in healthcare, diagnosis, and treatment accessibility.   At Smartway, supporting Rare Disease Day is extremely important to us. It highlights our mission and the importance of our role in helping patients access difficult-to-obtain medications, including #orphandrugs and #unlicensedmedicines. Using our global supply chain, regulatory expertise, and valuable partnerships, we ensure that medicines are delivered safely and on time.   On 29 February 2024, we came together to raise awareness of rare diseases, advocating for increased awareness, research, and support for individuals and families who are affected. We organised internal events to share information and start conversations about #rarediseases and the importance of raising awareness.   👉🏼 Read our latest BLOG to find out how Smartway helps patients with rare diseases access the therapies they need: https://lnkd.in/e2ncYmzM   #Pharmaceuticals #Pharma #MedicineAccess #EAP #EarlyAccessPrograms

February 29th is #RareDiseaseDay, a day to raise awareness about the ~7,000 rare diseases impacting patients all around the world. According to our Global Trends in R&D 2024 report, over the past five years, 144 drugs launched with orphan drug designations, representing 54% of the 267 launches and indicating a significant focus on innovative medicines for rare diseases. Download the report here: https://bit.ly/42WSfjs #RareDiseaseAwarenessDay #RareIs #RareWarrior

Selenite is not equivalent to selenium. It as its own specific properties. See references in cited review article below. 1- At low dose and concentration selenite (ie less than 500 microgram/day during few days, optimal selenium intake seems between 100-150 microgram/day) is a good Se donor, which is required for key antioxidant selenoenzymes (see below reference review articles). It is therefore a pro-antioxidant is that case. 2- But, selenite (Na2SeO3) should be also classified as an active ingredient (AI) (drug) depending on concentration (=> dose and speed of intravenous injection) (see below reference review articles especially table 1 of part B). Cytotoxic concentration is above 1-5 micro mole/L of Se under the form of selenite (Na2SeO3) (see figure in joined peace, graphical abstract, figure 2 and table 1 of part B review article below). Bolus injection of high dose selenite may reach cytotoxic concentration and then should be classified as chemotherapeutic intervention. In addition, it should be clearly specify if the dose is express in mg Se as selenite or in mg of selenite (Na2SeO3). There's a ratio of two to one between these two expressions. NOAEL: 400 microgram/day). Optimal daily intake may be for Se between 100-150 microgram/day (Rayman). AI reasons: 1- Selenite has been studied since long term as chemotherapeutic agent (see text and table 1 of part B, phase I Brodin O et al, 2015; Breuer O et al 2023 and the other with key words Brodin selenitpe) 2- It’s general lethal dose is similar to arsenic salt and its margin of safety is narrowed in sepsis. 3-It’s general toxicity mimic sepsis as documented in poisoning. 4- When its administration (e.g. rapid IV) allows reaching cytotoxic concentration in blood or tissues, selenite research should follow drug development process and not be directly studied in human. 5- Selenite is teratogenic, mutagenic and maybe carcinogenic. Human body content in Se in a repleted people is only 10-15 mg. However, there is an interest for developing a traitement based on selenite cytotoxicity. But that requires to follow drug development process. More details: Selenocompounds and Sepsis-Redox Bypass Hypothesis: Part B-Selenocompounds in the Management of Early Sepsis. Antioxid Redox Signal. Xavier Forceville, Pierre Van Antwerpen, Djillali Annane, and Jean Louis Vincent, 2022 Nov;37(13-15):998-1029. Or free beta access via pubmed (HAL) Below (attached )is a copyright-free figure focusing on selenite cytotoxicity. It is adapted from part B's forum review article published in Antioxidant and Redox Signaling (Fig 2).

#OBRxBrands #OurWorkWednesday OPZELURA is indicated for: – the topical short-term and non-continuous chronic treatment of mild to moderate atopic dermatitis in non-immunocompromised adult and pediatric patients 12 years of age and older whose disease is not adequately controlled with topical prescription therapies or when those therapies are not advisable. – the topical treatment of nonsegmental vitiligo in adult and pediatric patients 12 years of age and older. To Learn More: https://lnkd.in/eyksaJTW #OptiBrand #OptiBrandRx #OBRx #OWW #Pharma #PharmaMarketResearch #PharmaStrategy #Strategy #marketresearch #getmore #PharmaExperts #OPZELURA #Vitiligo

Treatments for rare diseases are becoming more and more common with half of US FDA approvals in 2023 receiving orphan designation. These treatments offer hope to many patients afflicted by previously untreatable terminal or degenerative conditions. But they are also very expensive on a per-patient basis, creating difficulties for payers. My colleagues Marc Tomassi and Francois Lucas offer their thoughts on four key strategies for pharma and biotech companies to help with access and pricing. https://lnkd.in/dgFx7Fxa

The GARDIAN Registry has released data on the first 26 patients: 96% with Gaucher disease Type 3 (GD3) and 4% with Type 2 (GD2). GARDIAN (Gaucher Registry for Development, Innovation, and Analysis of Neuronopathic Disease) is a global initiative by the International Gaucher Alliance (IGA). It aims to improve understanding of GD2 and GD3 by collecting personal experiences from patients and families. At Prevail Therapeutics, we celebrate the remarkable work of the IGA as we recognize the critical need to include patient and caregiver voices throughout the drug development process. By collaborating with the IGA to help support GARDIAN, we hope to develop a more authentic understanding of the patient experience that will improve current drug development efforts. Discover more about the results here: https://bit.ly/4eipC5w #GARDIANRegistry #GaucherDisease #InternationalGaucherAlliance #PatientDrivenResearch #InnovationInHealthcare