MILAN, 27 September 2018 – The final ceremony of the prestigious Prix Galien Italy 2018, considered a “Nobel Prize” in the field of pharmaceuticals and medical devices, was held this morning at the Rosa Grand Hotel. The event saw the participation of representatives of the major pharmaceutical companies operating in Italy.
The drugs awarded the Prize for innovation, by category, are: Ocrevus® by Roche for the category “Biological Drugs”, Spinraza® by Biogen for the category “Orphan Drugs”, Alofisel® by Takeda for the category “Advanced Therapies, ATMP”, Januvia® by MSD for the category “Real World Evidence”, RWE.
The drugs receiving Special mention are: Dupixent® by Sanofi for the category “Biological Drugs”, Vaxelis® by MSD for the category “Biological drugs”, Ilaris®, by Novartis for the category “Orphan Drugs”.
· DETAILS of the prize and special mention recipients and the GROUNDS for the award stated by the jury are reported below
· PHOTOS of the ceremony can be downloaded HERE
“Prix Galien is an initiative created in France in the 70s which aims to promote innovation in pharmaceutical research – explains Dr. Alessandro Gallo, managing director of Springer Healthcare. The Award takes place in various forms in about 20 countries and is coordinated by the Galien Foundation in New York. Springer is in charge of the Awards in Italy and Germany and, as of 2019, will also take care of the Dutch ceremony. In Italy the Prize has reached its 26th edition”.
Assessment of candidates to Prix Galien Italy is carried out by an independent Scientific Committee, chaired by Professor Pier Luigi Canonico, from the University of Eastern Piedmont “Amedeo Avogadro”. The committee carries out its assessments on a voluntary basis.
“Identifying innovative drugs is no doubt one objective of the pharmaceutical industry, but it is especially important for improving the health of patients – said Salvatore Cuzzocrea, Rector of the University of Messina and member of the Prix Galien Scientific Committee. However, one should avoid the risk of regarding as ‘innovative’ any new drug placed on the market”.
“Advances in biotechnology and regenerative medicine have definitely contributed to pharmaceutical innovation in recent years– stated, along the same lines, Annamaria De Luca, Full Professor of Pharmacology at the University of Bari “Aldo Moro” – but such innovation cannot and should not be restricted to the concept of innovative “product”; rather, it should be aimed […] at the possibility of treating unmet clinical needs”.
The 2018 edition of the Prix Galien features two major novelties.
The first is the activation of a prize reserved for advanced therapy medicinal products (ATMP), which include drugs for gene therapy, somatic cell gene therapy, tissue engineering products or combinations of the above.
The second novelty is the activation of a prize reserved for medical devices (aids, prostheses and orthoses), in collaboration with the Italian Society for Physical and Rehabilitation Medicine (SIMFER), whose representatives have joined the awards committee to evaluate the application files relating to their area of expertise. Awards for the category Medical Devices took place in Ancona on September 20, during the 46th National Congress of SIMFER; the winner is Vassilli S.r.l. from Padua, an Italian company that has developed an innovative verticalising electronic wheelchair.
The Real World Evidence (RWE) award, launched last year and confirmed in the current edition, is reserved for drugs subjected to post-approval studies able to confirm or redefine the efficacy and safety of a drug in real-world clinical settings or to contribute to the understanding of a disease through investigations on the drug.
The winners of the Prize reserved for research scientists aged under 35 working in Italy, are: Valerio Chiurchiù (Rome Bio-Medical Campus and IRCCS Santa Lucia Foundation in Rome) and Francesco Di Lorenzo (IRCCS Santa Lucia Foundation in Rome and Tor Vergata University of Rome).
DETAILS OF AWARD AND SPECIAL MENTION RECIPIENTS, AND THE GROUNDS FOR THE AWARD AS STATED BY THE JURY
Winner – Biological Drugs category
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 ocrelizumabproved 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
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.
“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
ALOFISEL® (darvadstrocel), indicated for the treatment of complex perianal fistulae in adult patients affected by Crohn’s disease.
“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
JANUVIA® (sitagliptin), indicated in adults with type 2 diabetes mellitus to improve glycaemic control, in monotherapy, dual therapy and triple oral therapy.
“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 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
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
VAXELIS® (hexavalent vaccine) for diphtheria, tetanus, pertussis, poliomyelitis, invasive diseases caused by Haemophilus influenzae type b (Hib) and hepatitis virus.
“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
ILARIS® (canakinumab), a fully human monoclonal antibody targeting interleukin 1_, which has undergone years of extensive clinical development for a variety of rare auto-inflammatory diseases.
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.”