Business Wire

CDKL5 Deficiency Disorder Voice of the Patient Report Submitted to FDA

Share

Understanding the patient voice is fundamental for therapy development. Companies need to ensure that what matters most to patients is being assessed, and regulators and payers asks for demonstration of value to support approval and reimbursement. Today, the Loulou Foundation and the International Foundation for CDKL5 Research (IFCR) are proud to release the “CDKL5 Deficiency Disorder Voice of the Patient Report”, capturing the voice of patients living with this rare genetic disease that affects thousands of people.

This press release features multimedia. View the full release here: https://www.businesswire.com/news/home/20200617005017/en/

The report is the outcome of a groundbreaking patient-centered meeting hosted in November 2019 as part of the FDA Patient Focused Drug Development initiative. The initiative was established by FDA to more systematically gather patients’ perspectives about their condition and available therapies. The meeting was attended by FDA representatives from CDER, CBER and the Office or Orphan Products among others.

Although there are more than 10,000 diseases, only a few dozen have been the subject of PFDD meetings. This includes CDKL5 Deficiency Disorder (CDD), a rare genetic disease that leads to frequent seizures shortly after birth and severe impairment in neurological development, with most affected people being unable to walk, talk or care for themselves.

The majority of the responders during the live meeting poll and post-meeting survey reported global developmental delay as the most devastating aspect of the disease. “In the beginning, all I wanted to do was stop the seizure,” explained Karen Utley, President of IFCR and mother to a 13-year old daughter with CDD. “However, as my daughter grew, it became crystal clear that this disorder is not simply an epilepsy. Accepting the reality of the situation and understanding that short of a treatment and a cure, your child will not develop into an independent adult, is a crushing blow.”

There is currently no therapy approved for the treatment of CDD, but there are several programs in clinical development and a growing interest from the drug development industry. “Celebrating this PFDD meeting was a big milestone for our field and one more step towards having approved and hopefully disease-modifying treatments,” said Ana Mingorance PhD, Chief Development Officer of the Loulou Foundation. “The timing was particularly important because there are four ongoing clinical trials as well as additional active preclinical programs that we hope will reach the clinic in the coming few years.”

The meeting and the report emphasize the urgent need for increased awareness of CDD, as well as the inadequacy of current treatments and lack of therapeutic options for the refractory seizures, global developmental delay and additional comorbidities associated with the disorder. By capturing the outcomes of this dialogue, the Voice of the patient Report will serve as a lasting resource for regulators, life science companies, researchers, the patient community, and the public.

CDD was also recently designated with a new disease code in the International Classification of Diseases (ICD), the medical classification list from the World Health Organization (WHO). These critical milestones serve as a testament to the power of patient advocacy groups working together toward shared goals of the CDD community. Both the PFDD meeting and the approval of a new ICD-10 code are the culmination of over a year of planning and collaboration by the groups that hosted the meeting.

The IFCR and Loulou Foundation are grateful to all meeting participants in person and via webcast, including the caregivers representing CDD symptoms, the physicians and scientific experts who participated, and the FDA for their support. They are also grateful to the caregivers that contributed to the post-meeting survey.

To read the report and learn more about the CDD EL-PFDD meeting, including a recording of the entire session, visit www.cdkl5.com/pfdd

About CDD
CDKL5 Deficiency Disorder (CDD) is a rare developmental and epileptic encephalopathy resulting from loss-of-function mutations in the CDKL5 gene. Presenting first as infantile spasms within the first weeks of life which progresses to intractable epilepsy, CDD patients also display profound neurodevelopmental delay, with generalized hypotonia, impaired motor skills, and severely impaired speech and vision. CDD patients also have impaired sleep, gastrointestinal function, and respiratory issues. With an incidence of approximately 1 in 40,000 live births, CDD is one of the most common monogenic pediatric epilepsies, with several thousand patients estimated in the US and Europe alone. No therapies exist to treat the neurodevelopmental symptoms of CDD, and the epilepsy associated with CDD is poorly managed by current anti-seizure medications.

About Loulou Foundation
Loulou Foundation is a private, non-profit foundation dedicated to therapeutic development for CDD. Founded in 2015, Loulou Foundation drives pre-clinical, translational, and clinical research on transformative therapeutics for CDD, with the goal of delivering meaningful treatments and eventual cures for CDD to patients and their families.
www.louloufoundation.org

About International Foundation for CDKL5 Research (IFCR)
IFCR is a US-based patient advocacy organization founded in 2009 by families from around the world affected by CDD. Since then, IFCR has funded groundbreaking research, including animal and cell model generation, patient-driven natural history, and a multidisciplinary clinical research network. Pioneering this network of CDKL5 Centers of Excellence across the United States has facilitated the development of a clinical research model that demands high-quality patient care, while accelerating clinical trial readiness. Above all, IFCR supports all CDKL5 families and caregivers by providing education, advocacy, and awareness.
www.cdkl5.com

Contact information

Daniel Lavery, PhD
Chief Scientific Officer, Loulou Foundation
Director, CDKL5 Program of Excellence, Orphan Disease Center
Perelman School of Medicine
University of Pennsylvania
T: +1 (215) 746-6725
E: dlavery@louloufoundation.org
www.louloufoundation.org

Karen Utley, BSN
Co-Founder and President
International Foundation for CDKL5 Research (IFCR)
E: kutley@cdkl5.com
www.cdkl5.com

About Business Wire

For more than 50 years, Business Wire has been the global leader in press release distribution and regulatory disclosure.

Subscribe to releases from Business Wire

Subscribe to all the latest releases from Business Wire by registering your e-mail address below. You can unsubscribe at any time.

Latest releases from Business Wire

Takeda Presents New Data Highlighting Scientific Advancements in Lung Cancer at ESMO Virtual Congress18.9.2020 13:55:00 EESTPress release

Takeda Pharmaceutical Company Limited (TSE:4502/NYSE:TAK) (“Takeda”) today announced that the company is presenting data from its lung cancer portfolio at the virtual European Society for Medical Oncology (ESMO) conference. Notably, insights from sub-analyses of the Phase 3 ALTA 1L study reinforce both the compelling evidence of intracranial efficacy with ALUNBRIG® (brigatinib) as a first-line treatment for patients with anaplastic lymphoma kinase-positive (ALK+) non-small cell lung cancer (NSCLC) as well as associated quality of life (QoL) data. Takeda is also featuring updated 10-month follow-up results from the Phase 1/2 trial of mobocertinib (TAK-788), demonstrating mobocertinib achieved a duration of response (DoR) of more than one year in the trial’s study population of patients with epidermal growth factor receptor (EGFR) Exon20 insertion+ metastatic NSCLC (mNSCLC). “We’re pleased to present our ongoing research in lung cancer at this year’s virtual ESMO congress, including new

Incyte Announces Encouraging Results From Phase 2 Trial of Retifanlimab (INCMGA0012) in Patients With Previously Treated, Advanced Squamous Cell Carcinoma of the Anal Canal18.9.2020 13:00:00 EESTPress release

Incyte (Nasdaq:INCY) today announced results from its Phase 2 POD1UM-202 trial evaluating retifanlimab, a PD-1 inhibitor, in previously treated patients with advanced squamous cell carcinoma of the anal canal (SCAC) who have progressed following standard platinum-based chemotherapy. The trial enrolled 94 patients, including those with well-controlled human immunodeficiency virus (HIV) infection (10%). Retifanlimab monotherapy resulted in a confirmed objective response rate (ORR) of 14% as determined by independent central review (ICR) using RECIST v1.1. Responses were observed regardless of PD-L1 status, presence of liver metastases, age or HIV+ status. Retifanlimab was generally well-tolerated with a safety profile as expected of a PD-1 inhibitor and no loss of HIV infection control. Key findings from POD1UM-202: N=94 ORR* (95% CI) 13.8% (7.6-22.5) Best OR*, n 1 CR 12 PR 33 SD DCR 48.9% DOR, median (95% CI), months 9.5 (5.6-NE) PFS, median (95% CI), months 2.3 (1.9-3.6) OS, median (95

Sigfox and Cube Infrastructure Managers Announce Major Partnership in IoT Infrastructure18.9.2020 10:30:00 EESTPress release

Sigfox, the global 0G network1 and cloud provider for industrial data, is proud to announce a new strategic alliance with Cube Infrastructure Managers (Cube), through the sale of its German 0G network to Cube. Sigfox has grown its 0G IoT services by rolling out 0G networks across 72 countries and regions, which was largely achieved with partners called Sigfox Operators. These operators are the owners of the 0G networks, which they operate as exclusive connectivity providers of Sigfox IoT services, offering worldwide connectivity to customers. The sale of the German network to Cube will allow Sigfox to finance its continued innovation efforts in data value extraction and improvements in cloud algorithms to reduce energy consumption and allow the implementation of even more cost-effective devices and sensors. This press release features multimedia. View the full release here: https://www.businesswire.com/news/home/20200918005116/en/ Cube Infrastructure Managers, the European infrastructu

Sensorion successfully raises approximately €31 (US$ 36.5) million in an oversubscribed private placement to US and European investors18.9.2020 09:00:00 EESTPress release

Regulatory News: Not for release, publication or distribution, directly or indirectly, in or into the United States, Canada, Australia or Japan. This press release is not intended as an offer and is for informational purpose only Sensorion (Paris:ALSEN) (FR0012596468 – ALSEN – the “Company”) a pioneering clinical-stage biotechnology company which specializes in the development of novel therapies to restore, treat and prevent within the field of hearing loss disorders announces today the success of its previously announced capital increase. The Company has placed 18,236,000 new ordinary shares with a nominal value of €0.10 each (the “New Shares”), for total gross proceeds of approximately € 31 million by means of an accelerated bookbuild offering to the benefit of categories of persons (the “Reserved Offering”). The issue price of the New Shares is €1.70 per share, representing a 3.5% discount to the weighted average share price on the day preceding the date on which the issuance price

ESMO 2020: Phase II CLARINET FORTE Results Show Increasing Dose Frequencies of Somatuline® Autogel® (lanreotide) Allows Patients with NETs to Delay Treatment Escalation by up to 8.3 Months18.9.2020 08:00:00 EESTPress release

Regulatory News: Ipsen (Euronext: IPN; ADR: IPSEY) today announced the release of first efficacy and safety data from the CLARINET FORTE study, with the abstract to be presented as a mini-oral presentation at the 2020 European Society for Medical Oncology (ESMO) Congress, taking place virtually from 19-21 September 2020. The prospective single-arm, open-label, exploratory, international Phase II study investigated the efficacy and safety of increasing the dose frequency of Somatuline® Autogel ® (lanreotide) in patients with pancreatic or midgut NETs with centrally-assessed progression within the last two years while on a standard lanreotide regimen for ≥24 weeks. An extension of progression-free survival (PFS) rates and encouraging disease-control rates (DCR) were recorded in both tumor types, with no new safety signals. “These results support a clinically meaningful benefit to a population of patients with high unmet medical need by potentially delaying escalation to more toxic treatm

Vifor Pharma Group Announces Successful Sale of OM Pharma18.9.2020 08:00:00 EESTPress release

Regulatory News: Vifor Pharma has today announced the successful sale of 100% of the share capital of OM Pharma, a Vifor Pharma Group company to Optimus Holding Ltd. The terms of the deal include: A purchase consideration of MCHF 435 for 100% of the share capital An earn out related to potential future value gains on 20% of Optimus Holding Ltd. equity to be determined before the end of 2027 upon a trade sale, IPO or EBITDA multiple This earn out together with the purchase consideration could result in a total transaction value exceeding MCHF 500. The deal is expected to close within 30 days. OM Pharma is a Geneva-based company mainly active in the field of microbial derived immunotherapeutics and has developed strongly outside the core strategy of Vifor Pharma over the past few years. Optimus Holding Ltd. is a Swiss Group, founded by Etienne Jornod together with long-standing Swiss entrepreneurs and Abdi Ibrahim (28.5%), a Turkey-based pharmaceutical company operating in 12 countries a

In our pressroom you can read all our latest releases, find our press contacts, images, documents and other relevant information about us.

Visit our pressroom