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This search function provides links to outputs produced by NIHR Innovation Observatory. These are briefing notes or reports on new or repurposed technologies. This search will not return all technologies currently in development as these outputs are produced as required for our stakeholders.

Innovation Observatory > Drugs

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Drugs

December 2017

Tocilizumab (RoActemra) for Adults with Systemic Sclerosis – Second Line

There is currently no cure for systemic sclerosis and treatment options focus on the management of symptoms. Tocilizumab is intended for the treatment of systemic sclerosis in adults. It is currently in development and early research indicates that it is able to target the key mechanisms thought to be responsible for the disease. If licensed, …

Drugs

December 2017

Lenadogene Nolparvovec (GS-010) for Vision Loss from Leber’s Hereditary Optic Neuropathy Due to Mutation of the ND4 Gene

There are currently very few treatment options for LHON. Most treatment methods are used to support the patient rather than cure them. Lenadogene nolparvovec is a new gene-targeted treatment intended for vision loss caused by LHON due to an alteration in a specific gene. As a genetic medicine, lenadogene nolparvovec could be a promising option …

Drugs

December 2017

Neridronic Acid (Nerixia) for Complex Regional Pain Syndrome

Currently there are no approved treatments for CRPS. Neridronic acid administered by injection is currently being developed to treat CRPS and early results indicate that it has the possibility to control the condition. If approved neridronic acid will be a potential new treatment for CRPS.

Drugs

December 2017

Upadacitinib for Adults with Moderate to Severe Active Rheumatoid Arthritis After Conventional Synthetic Disease-Modifying Anti-Rheumatic Drugs (DMARDs) or Biologic DMARDs Failure

There is currently no cure for rheumatoid arthritis and treatment aims to improve quality of life and to prevent or reduce joint damage. The most common treatment options are steroids to reduce inflammation, medications to reduce pain and swelling, and medications that slow the progression of joint damage from RA. Upadacitinib is currently being developed …

Drugs

December 2017

ALXN 1210 for Paroxysmal Nocturnal Haemoglobinuria – First Line

ALXN1210 is an antibody under development that prevents the destruction of red blood cells (RBCs). The drug is administered by intravenous injection that requires fewer dosing regimen compared to current treatment option. If ALXN1210 is licensed for use in the UK, it could represent an additional treatment option for patients with paroxysmal nocturnal haemoglobinuria.  

Drugs

December 2017

Lenalidomide (Revlimid) for Newly Diagnosed Multiple Myeloma – First Line

Lenalidomide in combination with bortezomib and dexamethasone is being developed for the treatment of adult patients with MM. While each of these drugs are already approved for treating a range of malignant blood diseases, the combination of the three drugs has shown significant potential particularly for the treatment of newly diagnosed MM. If approved, it …

Drugs

December 2017

Ponesimod for Relapsing-remitting Multiple Sclerosis

Ponesimod is a drug which works by blocking the signals which allow the body’s immune cells to travel to and damage the nerve cells. By preventing the immune cells from damaging the nerves, it is thought this drug will stop the damage which causes MS ‘relapses’. In clinical trials it has been shown that ponesimod …

Drugs

December 2017

IMCgp100 for the Treatment of Advanced or Metastatic Uveal Melanoma

There are currently few effective treatments available for advanced or metastatic uveal melanoma, including surgery to remove the cancer, radiotherapy or chemotherapy. IMCgp100 is a drug under development that would enable the body’s own immune system to recognise and specifically target and kill cancer cells.

Drugs

December 2017

Risankizumab (by subcutaneous injection) for Active Psoriatic Arthritis After Inadequate Control or Intolerance to Conventional Drugs

Risankizumab is a drug which is injected into the skin. It works in a unique way by blocking an important molecule in inflammation from a process which allows the body’s immune cells (specifically T-cells) to activate other immune cells and release substances which cause inflammation (cytokines).

Drugs

December 2017

Ribociclib and Fulvestrant and PI3K Inhibitor (Buparlisib or Alpelisib) for HR Positive, HER2 Negative Postmenopausal Breast Cancer – Second or Third Line

The combination of ribociclib, fulvestrant, and buparlisib or alpelisib is being developed for the treatment of HR+ and HER2- locally recurrent or advanced metastatic breast cancer. Ribociclib is given by mouth (capsules). It acts by helping to slow the progression of cancer by inhibiting two certain types of proteins that play a role in ensuring …

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