Expanded Access Program May Offer Drug Access to SCN2A Kiddos Prior to a Potential Drug Approval – Praxis has disclosed that they will be opening an Expanded Access Program (EAP) which may provide medication to patients while their drugs are still in clinical development (pre-FDA approval). Praxis has 1 medication potentially in clinical development for SCN2A (PRAX-562; a persistent sodium current blocker which could be beneficial for SCN2A gain-of-function variants) and two medications in pre-clinical development (1. PRAX-222 an antisense oligo (ASO) specific to SCN2A which could be beneficial to gain-of-function SNC2A variants, and 2. SCN2A-LOF (mechanism of action yet to be disclosed) that could potentially be beneficial to loss-of-function variants).
What are Expanded Access Programs (EAPs) – Prior to us families getting access to medications they typically need to be approved by the FDA. Two exceptions to this are 1. during clinical trials, and 2. when access is granted through an expanded or managed access program. The purpose of a clinical trial is to determine if both a drug is safe and effective. To accomplish this the patients allowed into the study are typically restricted to limit the variables of the trial and maximize the probability of success. Typically, EAPs and MAPs have more flexible criteria for eligibility relative to clinical trials.
What Should We as Caregivers Consider Before Applying for an EAP? – Drugs that are not approved by the FDA lack sufficient efficacy and or safety data to date to support FDA approval. This may just mean that more studies are required to prove the drug’s safety/efficacy profile, or it may mean that the drug is either not safe and/or not effective. Opting into a EAP means you are willing to accept the risk that the drug is not safe/effective to gain access earlier. Somethings that should be considered as caregivers include: 1. What are our goals for our kiddo and would this medicine help us get to that goal, 2. is my child in a stable enough position to wait for further development of this agent or others, 3. How strong is the clinical data to date for the medication in the EAP and how similar were the patients studied to my child, 4. Are there other options that make more sense to try first (such as other medications currently approved by the FDA), and 5. What does my child’s neurologist and primary care provider think?
How Would I Enroll into an EAP if Interested? – EAPs are typically accessed with the assistance of your child’s neurologist or other provider. Praxis’ eligibility criteria include: 1. Serious or life-threatening condition and is either no longer responsive to or no longer able to tolerate any available treatment option, and no comparable or satisfactory alternative treatment is available to treat the condition, 2. the medicine is in active clinical development with sufficient data available to and determine an appropriate dose and schedule for the patient’s specific condition (likely completed phase 1 study in SCN2A patients or currently in phase 2 studies in SCN2A), 3. a benefit-risk analysis, based on both the available clinical data as well as the requesting physician’s assessment of the individual patient’s condition and history, supports making the investigational drug available, 4. the patient is ineligible for participation in any ongoing clinical study of the investigational product, which includes lack of access due to geographic limitations, 5. making the investigational drug available will not negatively impact or delay the conduct of clinical trials or regulatory review or approval of the investigational drug for broader patient access, and 6. there is adequate supply of the investigational drug.
For More Detailed Information on the Praxis EAP - see https://praxismedicines.com/eap/
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