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Ongoing interactions between the US Food and Drug Administration (FDA) and CLINUVEL’s scientific teams continue to take place as the final outcome date of October 6, 2019 approaches. This date has alreayd been extended from May 31st to October 6th. In the latter half of the scientific review, the FDA will attempt to address substantive review issues which have not yet been covered by its committee members. At this stage, the FDA will use its time to gain a broader insight of risks and benefits while reserving the right to identify new review issues that may trigger an extension of the Prescription Drug User Fee Act (PDUFA) goal date. There is no indication that the current PDUFA date cannot be met; it is CLINUVEL’s expectation that October 6th will remain the current PDUFA goal date for an outcome to be provided.
Read Clinuvel's August Newsletter at…/20…/08/CLINUVEL-Communique-VI.pdf

Medical Moments
Acute Porphyria
Medical Moment: Do you know the benefits of Prophylactic Heme Treatment? Read the abstract from an article written by Porphyria Expert, Dr. Herbert Bonkovsky. Attachment to the full article included below.
Acute intermittent porphyria (AIP), an autosomal dominant inborn error of metabolism, is the most common and severe form of the acute porphyrias. Attacks of severe abdominal pain, often with hypertension, tachycardia, are cardinal features of AIP, often requiring hospital admissions. Frequent recurrent attacks of AIP, defined as > 3 attacks in one year, during which at least one attack requires intravenous heme therapy, are associated with significant morbidity, lost productivity, and health care burden. We report two patients with such frequent attacks of AIP, who have been managed with prophylactic heme therapy on a weekly basis. We describe results particularly in relation to symptom control, biochemical findings, health care costs, quality of life, and utilization of resources. During 11-month duration of weekly prophylactic heme infusions, we observed a 100% decrease in acute attacks and inpatient admissions in one subject and a 75% decrease in the other. During this time, we also observed a significant decrease in the number of emergency room visits. The decrease in number of acute attacks requiring hospital admission was associated with significantly decreased health care costs and improved quality of life. Reduction of both emergency room visits and hospital admissions decreased the utilization of health care services. Outpatient weekly infusions were also noted to be associated with better reimbursements and reduced overall costs of health care for the subjects. Both our subjects also endorsed better symptom control, quality of life and better understanding of disease. Thus, prophylactic heme therapy, through a multi-disciplinary approach, decreases the incidence of acute attacks, decreases health care costs and leads to better patient satisfaction and quality of life.
Medical Moment: Hepatitis C
According to experts - “Hepatitis C is common in PCT. In some areas where this viral infection is quite prevalent, especially in southern Europe and some parts of the U.S., as many as 80% of PCT patients are infected with this virus. How this particular virus contributes to developing PCT is not known. Other hepatitis viruses are seldom implicated.” If you have Hepatitis C, have PCT and you are interested in participating in research, please contact Edrin Williams, Director of Patient Services for information about the Harvoni Study!
Medical Moment: Do you have our CEP Informational Page? This page can be shared with your caregivers and healthcare providers as an educational tool. Please click on the link below to download! If you would like me to send you a hard copy via mail, please email me at - Edrin Williams, Director of Patient Services.
Link: Please add attached document (CEP One Pager)
Find Your Shadow 2019
Last week the APF sent the McKillop Family to the “Happiest Place on Earth” through our Shadow Jumpers program! This deserving family of five was able to take advantage of all the parks had to offer “VIP style”! With thoughtful planning, Morgan (EPP) was able to stay protected from the sun while having a once in a lifetime experience. Morgan especially loved the thrill rides and took total advantage of the disability fast pass, which reduced her waiting times in the sun and reflective pavement. The family also had a special VIP tour guide, Meagan, who ensured that Morgan was taken care of and always positioned out of the sun. Though the weather was mostly overcast, during the sunnier times the family was able to shadow jump through stores, they only had to break out the sun umbrella ONCE! The family suggests taking mid-day breaks in order to avoid the worst of the sun and regroup.
The APF was overjoyed to hear all about the McKillop’s vacation and thrilled to have been a part of making it happen! We are already looking forward to Find Your Shadow 2020!


  1. We hope that if you suffer from Porphyria that you feel free to leave any questions or comments so that we may assist you to find accurate answers!


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