European Union: European Medicines Agency's - Human Medicines: Highlights Of 2017

On January 23, 2018, the European Medicines Agency (EMA) has published its annual report titled 'Human medicines: Highlights of 201741. In the report, the EMA has published an overview of its key recommendations of 2017 regarding the authorization of new medicines and the safety monitoring of medicines.

Advances in medicines authorizations are essential for public health as they have the potential to improve treatment of diseases. In 2017, EMA recommended 92 medicines for marketing authorization. Of these, 35 had a new active substance, which has never been authorized in the European Union (EU) before. Many of these medicines represent a significant improvement in their therapeutic areas; they include medicines for children, for rare diseases and advanced therapies42. Amongst the 35 new active substances (NAS) that EMA recommended, 11 were new drugs and biologics to treat cancer, 05 to treat neurological disorders, 04 for infectious diseases, 04 for immunology/rheumatology, 03 for endocrinology, 02 each for Uroephrology, haematology, and dermatology, 01 for Pneumonology, and 01 for hepatology/gastroenterology class of drugs.

Table 1: List of New Active Substances Approved by the European Medicines Agency

Sr. No. Medicine Approved Therapeutic Area
1. Axumin (Fluciclovine) Cancer/Oncology
2. Bavencio (Avelumab) Cancer/Oncology
3. Besponsa (inotuzumab ozogamicin) Cancer/Oncology
4. Fotivda (Tivozanib) Cancer/Oncology
5. Kisqali (Ribociclib) Cancer/Oncology
6. Lutathera (Lutetium (177Lu) oxodotreotide) Cancer/Oncology
7. Rydapt (Midostaurin) Cancer/Oncology
8. Tecentriq (Atezolizumab) Cancer/Oncology
9. Tookad (Padeliporfin) Cancer/Oncology
10. Varuby (Rolapitant) Cancer/Oncology
11. Zejula (Niraparib) Cancer/Oncology
12. Brineura (Cerliponase alfa) Neurology
13. Ocrevus (Ocrelizumab) Neurology
14. Oxervate (Recombinant human Nerve Growth factor (rhNGF)) Neurology
15. Reagila (Cariprazine hydrochloride) Neurology
16. Spinraza (Nusinersen) Neurology
17. Maviret (Glecaprevir/pibrentasvir) Infections
18. Prevymis (Letermovir) Infections
19. Trumenba (Meningococcal group B vaccine (recombinant, adsorbed)) Infections
20. Vosevi (Sofosbuvir / Velpatasvir / Voxilaprevi) Infections
21. Kevzara (Sarilumab) Immunology/Rheumatology
22. Spherox (Spheroids of human autologous matrix-associated chondrocytes) Immunology/Rheumatology
23. Tremfya (Guselkumab) Immunology/Rheumatology
24. Xeljanz (Tofacitinib) Immunology/Rheumatology
25. Crysvita (Burosumab) Endocrinology
26. Ozempic (Semaglutide) Endocrinology
27. Xermelo (Telotristat etiprate) Endocrinology
28. Lokelma (Sodium zirconium cyclosilicate) Uro-nephrology
29. Veltassa (Patiromer sorbitex calcium) Uro-nephrology
30. Adynovi (Rurioctocog alfa pegol) Haematology
31. Refixia (Nonacog beta pegol) Haematology
32. Dupixent (Dupilumab) Dermatology
33. Kyntheum (Brodalumab) Dermatology
34. Alofisel (Expanded human allogeneic mesenchymal adult stem cells extracted from adipose tissue) Hepatology/Gastroenterology
35. Fasenra (Benralizumab) Penumology

EMA's Special Program Approvals

EMA, in its report, also highlighted its expedited programs and incentives to bring products to market more quickly and spur development of products to treat rare diseases or other areas of high unmet medical need.

Orphan Medicines

To qualify for agency's orphan designation, a medicine must meet a number of criteria43:

  • It must be intended for treatment, prevention or diagnosis of a disease that is life-threatening or chronically debilitating;
  • The prevalence of the condition in the EU must not be more than 5 in 10,000 or it must be unlikely that marketing of the medicine would generate sufficient returns to justify the investment needed for its development;
  • No satisfactory method of diagnosis, prevention or treatment of the condition concerned can be authorized, or, if such a method exists, the medicine must be of significant benefit to those affected by the condition.

In 2017, the agency approved 19 drugs in its Orphan Medicine Program, out of which 13 were New Active Substances.

Accelerated Assessment

Accelerated assessment reduces the timeframe for the EMA's Committee for Medicinal Products for Human Use (CHMP) to review a marketing authorization application. Applications may be eligible for accelerated assessment if the CHMP decides that the product is of major interest for public health and therapeutic innovation44. This mechanism is reserved for medicines that are able to address unmet medical needs. It allows for faster assessment of eligible medicines by EMA's scientific committees (within up to 150 days rather than up to 210 days).

In 2017, seven medicines received a recommendation for marketing authorization following an accelerated assessment out of which five were new active substances.

Conditional marketing authorizations

The EMA supports development of medicines that address unmet medical needs of patients. In the interest of public health, applicants may be granted a conditional marketing authorization for such medicines where the benefit of immediate availability outweighs the risk of less comprehensive data than normally required, based on the scope and criteria defined in relevant legislation and guidelines45.

In 2017, three medicines received a recommendation for conditional marketing authorization out of which two were new active substances.

Approval under exceptional circumstances

Approval under exceptional circumstances is an agency's regulatory pathway that allows patients access to medicines that cannot be approved under a standard authorization as comprehensive data cannot be obtained, either because there are only very few patients with the disease, or the collection of complete information on the efficacy and safety of the medicine would be unethical, or there are gaps in the scientific knowledge. These medicines are subject to specific post-authorization obligations and monitoring46.

In 2017, two medicines were authorized under exceptional circumstances.

Table 2: Number of Drugs Approved by EMA Special Program Approvals

Program or Designation New Active Substances Approved Total Approvals
Orphan Medicine 13 19
Accelerated Assessment 05 07
Conditional Marketing Authorization 02 03
Approval Under Exceptional Circumstances 01 02

New uses for existing medicines

51 extensions of indication were recommended in 2017. The extension of the use of a medicine that is already approved in a new therapeutic indication can also offer new treatment opportunities for patients.

EMA's Safety Monitoring

Once a medicine is placed on the market, EMA and the EU Member States continuously monitor the quality and the benefit/risk balance of the medicine under its authorized conditions of use. In 2017, EMA gave new safety advice to manage risks observed with a number of medicines on the market in the EU. Regulatory measures ranged from a change in the product information to the suspension or withdrawal of a medicine to even recall of a limited number of batches.

Important new safety advice issued in 2017 included:

  • Information about a potential increased risk of lower limb amputation (mostly affecting the toes) in patients taking the SGLT2 inhibitors canagliflozin, dapagliflozin and empagliflozin used for type 2 diabetes.
  • Recommendations to restrict the use of some linear gadolinium agents used in MRI body scans and suspend the authorizations of others. EMA's scientific review found that small amounts of gadolinium may remain in the brain after a scan with these agents, although there is currently no evidence that these small amounts cause any harm.
  • Recommendation to restrict the use of Zinbryta for treating multiple sclerosis, in view of the risk of serious liver damage in some patients.
  • Recommendation to suspend marketing of paracetamol medicines designed to release the active ingredient over a long period (modified-release medicines) because of the difficulty in managing overdoses.
  • New recommendation on medicines containing a combination of dienogest 2 mg and ethinylestradiol 0.03 mg which can continue to be used to treat moderate acne when certain other treatments have failed, but should only be used in women who also choose oral contraception.
  • Prescription information for the antibiotic vancomycin to be changed to ensure appropriate use in the treatment of serious infections caused by Gram-positive bacteria. The recommendation aims to ensure appropriate use in the context of the fight against antimicrobial resistance.
  • New contraindication for Uptravi, which must not be taken simultaneously with medicines like gemfibrozil which are strong blockers (inhibitors) of the liver enzyme CYP2C8.
  • New recommendation for Symbioflor 2 to continue to be used to treat irritable bowel syndrome (IBS), but not for other functional gastrointestinal disorders. The company will provide a study on effectiveness and safety among patients with different features of IBS.
  • eview of human factor VIII medicines authoriszd in the EU - EMA concluded that there is no clear evidence of a difference in the risk of inhibitor development between the two classes of factor VIII medicines. Patients should therefore continue to use their factor VIII medicines as prescribed by the doctor.
  • ew contraception recommendations for male patients regarding concerns that mycophenolate medicines (used to prevent rejection of transplanted organs) cause miscarriages or birth defects.
  • New recommendation on injectable methylprednisolone medicines (used to treat the symptoms of severe allergic reactions) containing lactose which must not be used in patients with a known or suspected allergy to the proteins in cow's milk.


In 2017, the EMA saw an increase in the number of new/novel drugs and biologics it recommended for marketing authorization. New Drugs/ Novel drugs are innovative products that serve previously unmet medical needs or otherwise significantly help to advance patient care and public health.








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