With International SCN2A Awareness Day fast approaching, we are reflecting on the progress made last year and turning our attention to the new year filled with hope and possibility.
Despite the challenges of 2021, the FamilieSCN2A Foundation thrived.
› The SCN2A Clinical Trial Readiness Study began paving the way for new treatments for SCN2A-related disorders approved by the FDA.
› A virtual FDA ‘Listening Session’ was hosted allowing several families to share what it’s really like to live with SCN2A-related disorders with those who determine the approval of new treatments and cures.
› Virtual Table Talks Series were held allowing families unprecedented access to professionals.
› We continued to provide financial assistance for SCN2A families in need.
In 2022, our focus will be on BUILDING.
Since our inception in 2015, we have been steadily constructing a strong foundation that is anchored by our dedication to:
› COMMUNITY
› ADVOCACY
› RESEARCH
As winners of the 2021 CZI: Rare As One Grant, we will be able to build upon this already strong foundation. Over the next three years, we will receive $600,000 in funding and access to a network of CZI resources to strengthen our organizational capacity, grow our community, and advance international collaborative research toward shared priorities.
Together we will build toward a cure!
Frequently Asked Questions
Drugs typically have to clear multiple clinical trial hurdles in order to support approval. The typical trial phases are 1, 2, and 3 with lower number being earlier trials and larger numbers being more advanced trials.
Phase 1 trials are typically in healthy volunteers and are evaluating initial safety, and dosing in humans. These trials help ensure that the drug is safe enough to be evaluated in patients and also are used to identify an appropriate dosing range for subsequent trials.
Phase 2 trials are typically used to provide proof of concept (that the drug has an efficacy signal, is providing a benefit to patients) and also tests safety in the patient population. These trials often are used to help design and power registrational phase 3 trials.
Phase 3 trials are often the trials used to support the approval of the agent and may be the final clinical hurdle a drug needs to pass in order to be approved by the FDA.
This is a designation granted by the FDA when a drug is being developed to treat a pediatric condition that is serious and life-threatening and occurs in under 200k people in the US.
This is a program used by the US government to incentivize drug development for pediatric rare diseases. If a drug is approved that has been granted the rare pediatric disease designation, then the developing company receives a voucher that grants priority review of a future drug (which can accelerate the developmental time of a drug by ~4 months; for example: if drug#1 is approved and had been granted the rare pediatric disease designation then said company would receive a priority review voucher that they could use on drug#2 ).
This is a designation granted by the FDA when a drug is being developed to treat a rare disease (occurs in under 200k people in the US).
This is a program used by the US government to incentivize drug development for rare diseases. Drugs awarded this designation received a number of incentives by the FDA including: 1. Market exclusivity for 7 years post approval, 2. A waiver of application of user-fees, and 3. A 50% tax credit for clinical testing expenses