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New forms of RUCONEST® administration and additional products

With validation secured from the approval of the first product from our transgenic platform, we will now seek to initiate new projects on this platform.

Our transgenic platform remains the only technology that to date can deliver recombinant versions of certain complex human proteins in an economically viable way.

Our strategy:

  • New forms of administration for RUCONEST® for HAE
  • Clinical development of protein replacement products in Pompe and Fabry diseases
  • Expansion of rhC1INH clinical development in pre-eclampsia, contrast induced nephropathy (CIN) and other potential large indications
rhC1INH (RUCONEST®)

rhC1INH (RUCONEST®)

Recombinant human C1 inhibitor (rhC1INH) is a recombinant human C1 inhibitor protein. Natural C1 inhibitor DNA from a human source is used in Pharming’s protein production technology to ensure expression of the C1 inhibitor protein. Human C1 inhibitor is a human protein involved in the regulation of the complement and contact parts of the immune system. It is a controlling protein in early stages of these cascades and in that way a regulating factor of the immune system. 

By inhibiting these systems, the abnormal inflammation can be controlled. This is also the mechanism by which an HAE attack can be treated with C1 inhibitor

Prophylaxis of HAE

In acute HAE, each individual attack is treated. However, with prophylactic therapy, the patient is treated on a regular basis with the aim of preventing attacks or at least reducing the frequency of attacks. In July 2016, Pharming announced positive results from a Phase II clinical study exploring the use of RUCONEST® for prophylaxis of HAE.

Following the positive phase II study, the FDA invited Pharming to submit the phase II trial early. Subsequently, the FDA issued Pharming with a complete response letter in September 2018.

New formulation development of RUCONEST®

The “RUCONEST® liquid” formulation (from 14ml to 3ml) can be used as starting material for the generation of subcutaneous, intra-muscular & intradermal application systems.

New proprietary ‘painless’ intradermal delivery applications are being developed. A dissolving point device and a reservoir device. 

Example of possible injection methods

These painless versions should differentiate RUCONEST® from competitors, all of whom have painful injections.  “Painless” Nano-injections are the future for the treatment of both acute and prophylaxis HAE attacks and could be the future treatment of other indications in our pipeline.

New activities with rhC1INH (RUCONEST®)

Initial Therapeutic Indications selected:

  • Organ damage after contrast media application: Contrast-induced Nephropathy (CIN) recent positive results investigator-sponsored study. 
  • Tissue Damage after Toxic Event: Pre-Eclampsia; study to start Q1 2019
  • Tissue Damage after Hypoxic Event: Delayed Graft Function (new Investigator initiated study)
  • Shock response after trauma: Hypovolemic Shock– ongoing preclinical research projects with the US Army and US Air Force

Brand New IP:

  • New Pharming patents filed in 2018 covering the new indications
  • Patents cover all forms of rhC1INH
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