For healthcare professionals

Our commitment to you

Akcea Therapeutics UK strives not only to be an advocate for patients living with serious and rare diseases, but also to be a partner to the healthcare professionals who care for these patients. At Akcea Therapeutics UK, we believe our therapies have the potential to address the unmet needs and complex medical challenges of patients with these important, less understood disorders.

Our products

Our current products include drugs with the potential to treat patients with disorders that other types of drugs, such as small molecules and antibodies, have not been able to adequately address.

While traditional drugs modify proteins involved in diseases, our treatments decrease the production of these proteins in the first place, offering the potential to significantly improve how serious and rare disorders are addressed.1

Akcea Therapeutics has two drugs licensed in the EU. Both products are from Ionis Therapeutics’ antisense technology platform, were discovered by Ionis, and were either developed by Ionis or co-developed by Ionis and Akcea.

Tegsedi_R_EU_Triangle_RGB

In the UK, TEGSEDI is the first licensed and reimbursed therapy for the treatment of adult patients with stage 1 and 2 polyneuropathy caused by hereditary transthyretin amyloidosis (hATTR).2

The phase 3 study, called NEURO-TTR, demonstrated benefit compared with placebo across both primary end points of the study: the Norfolk Quality of Life Questionnaire-Diabetic Neuropathy (Norfolk QoL­DN) and the modified Neuropathy Impairment Score +7 (mNIS+7) at 66 weeks of treatment.2

Treatment with our product was associated with statistically significant improvements compared with placebo in measures of both neuropathy and quality of life (QoL).2

Two key safety risks were identified by the study: thrombocytopenia and renal impairment including glomerulonephritis. Enhanced monitoring was implemented during the study to support early detection and management of these issues.2

For important safety information on TEGSEDI, including method of administration, special warnings, drug interactions, and adverse drug reactions, please see the UK Summary of Product Characteristics (SmPC), which is available on the EMA website at https://www.ema.europa.eu/en/medicines/human/EPAR/tegsedi.

Next steps

In May 2019, we announced that the National Institute for Health and Care Excellence (NICE) has published its final Highly Specialised Technologies (HST) guidance for TEGSEDI for the treatment of stage 1 or stage 2 polyneuropathy in adult patients with hATTR.

For more information on hATTR, please contact the National Amyloidosis Centre (NAC), based at the Royal Free Hospital, London.

View the TEGSEDI Prescribing Information.

Download

 

Results of clinical trials with inotersen sponsored by Akcea Therapeutics are available from:
NCT01737398
NCT04270058

 

Adverse events should be reported. Reporting forms and information can be found at https://yellowcard.mhra.gov.uk/ Adverse events should also be reported to Akcea Therapeutics UK Ltd:
Email: MedInfoUK@akceatx.com
Telephone: +44 (0) 330 159 0174

WAY-Logo_BlackTriangle_EUsite

In May 2019, Waylivra became the second Akcea Therapeutics product to receive marketing authorisation from the European Medicines Agency (EMA). Waylivra is approved for use as an adjunct to diet in adults with genetically confirmed familial chylomicronaemia syndrome (FCS) at high risk of pancreatitis, who have responded inadequately to diet and triglyceride-lowering therapy.3

The phase 3 study, called APPROACH, met its primary end point of triglyceride reduction and showed a statistically significant lowering of the incidence of pancreatitis in patients with FCS at high risk of pancreatitis.3

The most common adverse reactions in the APPROACH study were injection site reactions and reductions in platelet levels.3

For important safety information on Waylivra, including method of administration, special warnings, drug interactions, and adverse drug reactions, please see the UK SmPC, which is available on the EMA website at https://www.ema.europa.eu/en/medicines/human/EPAR/waylivra.

Next steps

Akcea Therapeutics UK is engaging with NICE to ensure access to Waylivra for patients with FCS throughout the UK. Early access for eligible patients with FCS has been available through the Early Access to Medicines Scheme (EAMS).

View the Waylivra Prescribing Information.

Download

 

Clinical Trials:

Results of clinical trials with volanesorsen sponsored by Akcea Therapeutics are available from:
NCT02211209
NCT02300233
NCT02910635

 

Adverse events should be reported. Reporting forms and information can be found at https://yellowcard.mhra.gov.uk/ Adverse events should also be reported to Akcea Therapeutics UK Ltd:
Email: MedInfoUK@akceatx.com
Telephone: +44 (0) 330 159 0174

References:  1. Visser ME, Witztum JL, Stroes ESG, Kastelein JJP. Antisense oligonucleotides for the treatment of dyslipidaemia. Eur Heart J. 2012;33(12):1451-1458. doi:10.1093/eurheartj/ehs084.  2. TEGSEDI [summary of product characteristics]. Akcea Therapeutics Ireland Ltd; March 2019.  3. Waylivra [summary of product characteristics]. London, England: Akcea Therapeutics Ireland Ltd; 2019.