Winner – Biological Drugs category 

Roche candidate:

OCREVUS® (ocrelizumab), approved by the European Union for the treatment of adult patients affected by relapsing and primary progressive multiple sclerosis (MS). It is a humanised monoclonal antibody designed to cause selective depletion of B lymphocytes expressing the CD20 receptor, involved in the pathophysiology of multiple sclerosis, while preserving the capacity for B lymphocyte reconstitution and pre-existing humoural immunity.

The grounds for the award as stated by the jury

“Ocrelizumab is a biotechnological therapy with documented efficacy and clinical safety, which has been designated a “breakthrough therapy” by the FDA. In recent studies conducted on adult patients with relapsing MS, ocrelizumab proved able to reduce relapses by about half compared to high-dose interferon 1b, a standard treatment for the disease. Moreover ocrelizumab is the first drug to have shown efficacy in adult patients with primary progressive MS. In both disease forms ocrelizumab proved to have a favourable safety profile. The availability of a new drug indicated for both relapsing and primary progressive forms of MS is of paramount importance, given that all of the treatments currently offered for MS still leave areas of unmet needs in terms of treatment efficacy and safety.”

Winner – Orphan Drugs category 

Biogen candidate:

SPINRAZA® (nusinersen), the only disease-modifying drug indicated for 5q spinal muscular atrophy (SMA), a rare autosomal recessive neuromuscular disorder that mainly affects children and characterised by motor neuron degeneration.

The grounds for the award as stated by the jury

“Nusinersen obtained designation as an orphan drug by the FDA and EMA, and the two Regulatory Authorities also evaluated the application for accelerated assessment, recognising the importance of expediting access to therapy. Subsequently, AIFA recognised that the drug was highly innovative in its indication and authorised its reimbursement for the treatment of all types of SMA.

Its use has dramatically changed the course of the disease, achieving unexpected clinical endpoints in a disease for which no treatment has been available until now. Nusinersen considerably improves the patients’ life expectancy and quality of life, as well as impacting caregiver activity and the direct and indirect costs of the disease. The drug’s safety has been evaluated in a wide range of infants and children with SMA in controlled and open studies. Clinical development of the drug took place with the participation of a group of Italian clinicians with excellent expertise in the field of neuromuscular disorders.”

Winner – Advanced therapy medicinal products (ATMP) category  

 Takeda candidate:

ALOFISEL® (darvadstrocel), indicated for the treatment of complex perianal fistulae in adult patients affected by Crohn’s disease.

The grounds for the award as stated by the jury

“This is the first therapy based on adipose-derived mesenchymal stem cells to receive central marketing authorisation in Europe. Its peculiar mechanism of action and its status as an orphan drug (confirmed by the COMP, Committee for orphan medicinal products, Ed.) qualify it as an innovative drug. The available studies allow us to assess positively the risk-benefit profile for the use of this drug which, compared to the control arm, is associated with higher remission rates and lower relapse rates, related to an improved quality of life associated with the level of severity of the perianal disease.”

Winner – Real World Evidence (RWE) category 

MSD candidate:

JANUVIA® (sitagliptin), indicated in adults with type 2 diabetes mellitus to improve glycaemic control, in monotherapy, dual therapy and triple oral therapy.

The grounds for the award as stated by the jury

“Progenitor of an innovative class of drugs (dipeptidyl peptidase 4-inhibitors), sitagliptin has confirmed its real world versatility in combination therapies with other diabetes mellitus drugs, without causing any increase in side effects. Sitagliptin has in fact a superior safety and tolerability profile compared to other blood glucose-lowering treatments, which translates into improved treatment adherence, less discontinuation, less risk of admissions for cardiovascular or metabolic reasons and, consequently, lower costs for the health system. Italian research groups gave major contributions in both the drug’s clinical development phase and in the real-world studies.”

Winner – Medical Devices category 

Vassilli S.r.l. candidate:

18.70A BV50 – HI-LO Vario Avanti electronic verticalising wheelchair, a technological aid capable of verticalising, tilting and reclining the backrest and footrest in an anthropometric and independent manner thanks to its ability to memorise ideal positions.

The grounds for the award as stated by the jury

“The aid achieves an excellent balance between functional and postural needs, but also articular hygiene, personalisation and safety. It’s the result of an accurate design, which addressed clinical, anthropometric, kinematic and risk-management aspects. Intelligent electronics can record the users’ movements and make them functional to their needs or to those of the caregivers; this helps to achieve the goals of rehabilitation and reduce situations of danger. The aid offers an innovative contribution compared to the products currently on the market, and it aims to promote the concept of inclusion, by enabling the disabled person to take part in the community’s decisional and planning processes.”

Special mention (n. 1) – Biological Drugs category

 Sanofi candidate:

DUPIXENT® (dupilumab), recombinant human IgG4 monoclonal antibody indicated in the treatment of moderate-to-severe atopic dermatitis of the adult.

The grounds for the special mention as stated by the jury

“Dupilumab is the first biotechnological drug that has clinical efficacy in moderate-to-severe atopic dermatitis; its originality and innovativeness also lie in its pharmacological target. Five clinical studies showed very positive results on all the efficacy parameters evaluated, associated with a good tolerability profile. Its benefits in terms of itching and quality of life are well documented and important. The expected medical-scientific impact of this drug is very strong, given that the current pharmacological treatments for severe atopic dermatitis are unsatisfactory. The robustness of the clinical trial data and the patients’ need for this treatment are the elements that prompted AIFA to include this drug in the list of innovative drugs. It is, in fact, the first biological drug indicated for a non-oncological skin disorder to fulfil the Agency’s criteria for a truly innovative drug.”

Special mention (n. 2) – Biological Drugs category 

MSD candidate:

VAXELIS® (hexavalent vaccine) for diphtheria, tetanus, pertussis, poliomyelitis, invasive diseases caused by Haemophilus influenzae type b (Hib) and hepatitis virus.

The grounds for the special mention as stated by the jury

“The innovative formulation of the hexavalent vaccine, the only one available in Europe to contain an antigen component for pertussis (acellular) made up of 5 antigens, benefits from the clinical experience of immunogenicity, efficacy and safety previously acquired with the single antigens, and a ready-to-use format. It is a fully liquid vaccine, contained in a single pre-filled Luer-lock syringe, which allows for safer handling and reduces the risk of possible needlestick injuries or product contamination. Moreover it requires a shorter preparation time compared to vaccines to be reconstituted, with a lower risk of error.

Universal paediatric vaccination (against diphtheria, tetanus, pertussis, poliomyelitis, invasive diseases caused by Hib and hepatitis B) is a public health priority and is provided for by almost all national European vaccination schemes. This vaccine represents a further prevention tool for the benefit of children and health professionals.”

Special mention  – Orphan Drugs category

 Novartis candidate:

ILARIS® (canakinumab), a fully human monoclonal antibody targeting interleukin 1β, which has undergone years of extensive clinical development for a variety of rare auto-inflammatory diseases.

The grounds for the special mention as stated by the jury

Canakinumab is highly innovative in that it is the only drug approved for three rare auto-inflammatory diseases: TRAPS (tumour necrosis factor receptor-associated periodic syndrome), MKD (mevalonate kinase deficiency) and FMF (familial Mediterranean fever).

“Despite their being genetic disorders, canakinumab demonstrated that it can effectively treat the signs and symptoms of inflammation, and thus prevent the damage caused by the protracted and recurrent inflammation typical of these diseases, providing patients with: (1) rapid and prolonged control of acute exacerbations, (2) no or only minimal disease activity, (3) improved quality of life in terms of physical, mental and psychosocial health.”