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Survey about achondroplasia
We have designed a questionnaire with the purpose to better know the population connected to achondroplasia.
The questionnaire is optional and anonymous. The data obtained will be used for statistical purposes and to better understand the natural history of achondroplasia. Please consider answering.
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Phase 1 Study to Evaluate The Pharmacokinetics, Safety, And Tolerability Of Single Or Multiple Subcutaneous Doses of Recifercept is now active. 18 healthy adults will receive Recifercept, an innovative medicine for achondroplasia and the study will be ongoing for 1 year. This is the first step in the evaluation of the safety of the drug in individuals.
After this phase 1 study, if the drug is confirmed to be safe and tolerable and after the evaluation of the onset, duration, and intensity of a drug's effect in the human organism is assessed, the next step can be taken. That will be phase 2, in which children with achondroplasia with ages between 0 to 10 years-old will be invited to participate in a clinical trial.

Why Pfizer is conducting another phase 1 for Recifercept (or PF-07256472)?

Back in 2018, when Therachon was leading the research on TA-46 (now Recifercept), one Phase 1 was conducted. You can read more about this research here. Now, another phase 1 is starting and the question of why this repetition arises.
Pfizer developed a new formulation of TA-46, now named PF-07256472, which is purified further and is a dry powder for solution for injection. But beyond that, Pfizer wants to explore which will be the best daily dosing. So in this new Phase 1, the company is going to test different doses and different administration frequencies and not only once injection per week. It will be tested low and high doses and also placebo and different intake frequency. 

Why a purification process of TA-46 was done?

Purification means the removal of unwanted impurities present in an organic compound. Almost everything that we see these days is impure. The water we drink and the food we eat also need to go through levels of purification processes. Similar is the case with organic compounds. There are several methods of purification of organic compounds [2]. 

Dreambird study

Currently, more than 200 children around the globe are participating in the Dreambird study, the Observational Study Investigating Clinical & Anthropometric Characteristics of Children With Achondroplasia.  Know more about Recifercept (TA-46) mechanism is the animal model here.

 

phase 1 recifercept


Image 1: Summary of phase 1 clinical study. Credits: Clinicaltrials.gov

Phase 2 with children with achondroplasia may start around the 2nd quarter of 2021, yet to step into this stage, relevant and positive results of the current phase 1 are needed.

References:
Clinicaltrial.gov

2. Scilearn 

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