Table 2.

Innovativeness status of medicinal products in Italy

Medicinal product	Pharmacotherapeutic group	Therapeutic indication	Orphan	Status
Alecensa® (alectinib) Roche	Antineoplastic agents	As monotherapy for the treatment of adult patients with anaplastic lymphoma kinase‐positive advanced nonsmall cell lung cancer (NSCLC) previously treated with crizotinib.	No	Conditionally innovative
		As monotherapy for the first‐line treatment of adult patients with anaplastic lymphoma kinase‐positive advanced NSCLC	No	Fully innovative
Bavencio® (avelumab) Merck Serono	Antineoplastic agents	As monotherapy for the treatment of adult patients with metastatic Merkel cell carcinoma.	Yes	Conditionally innovative
Besponsa® (inotuzumab ozogamicin) Pfizer	Antineoplastic agents	As monotherapy for the treatment of adults with relapsed or refractory CD22‐positive B cell precursor acute lymphoblastic leukaemia. Adult patients with Philadelphia chromosome positive relapsed or refractory B cell precursor acute lymphoblastic leukaemia should have failed treatment with at least 1 tyrosine kinase inhibitor.	Yes	Conditionally innovative
Darzalex® (daratumumab) Janssen Cilag	Antineoplastic agents (monoclonal antibodies)	As monotherapy for the treatment of adult patients with relapsed and refractory multiple myeloma, whose prior therapy included a proteasome inhibitor and an immunomodulatory agent and who demonstrated disease progression on the last therapy.	Yes	Non‐innovative
		In combination with lenalidomide and dexamethasone, or bortezomib and dexamethasone, for the treatment of adult patients with multiple myeloma who have received at leastone prior therapy.	Yes	Fully innovative
Dupixent® (dupilumab) Sanofi‐Aventis	Immunosupressants	Treatment of moderate‐to‐severe atopic dermatitis in adult patients who are candidates for systemic therapy.	No	Fully innovative
		(The assessment of innovativeness refers to the following reimbursed indication only: adult patients with severe atopic dermatitis who are eligible for treatment with systemic therapy, for whom treatment with cyclosporin is contraindicated, ineffective or not tolerated.)
Hemlibra® (emicizumab) Roche	Antihemorrhagics	Routine prophylaxis of bleeding episodes in patients (all age groups) with haemophilia A with factor VIII inhibitors.	No	Fully innovative
Humira® (adalimumab) Abbvie	Immunosupressants	Treatment of paediatric chronic non‐infectious anterior uveitis in patients aged >2 years who have had an inadequate response to or are intolerant to conventional therapy, or in whom conventional therapy is inappropriate.	No	Conditionally innovative
Ibrance® (palbociclib) Pfizer	Antineoplastic agents	Treatment of hormone receptor (HR) positive, human epidermal growth factor receptor 2 (HER2) negative locally advanced or metastatic breast cancer: in combination with fulvestrant in women who have received prior endocrine therapy.	No	Non‐innovative
		Treatment of HR positive, HER2 negative locally advanced or metastatic breast cancer: in combination with an aromatase inhibitor.	No	Conditionally innovative (follower)
Ilaris® (canakinumab) Novartis	Interleukin inhibitors	Periodic fever syndromes in adults, adolescents and children aged 2 years and older:	No	Conditionally innovative
		‐Tumour necrosis factor receptor associated periodic syndrome (TRAPS)
		‐Hyperimmunoglobulin D syndrome (HIDS)/mevalonate kinase deficiency (MKD)
		‐Familial Mediterranean fever (FMF), in combination with colchicine, if appropriate (for this indication the assessment of innovativeness refers to the following reimbursed indication only: patients with FMF for whom treatment with colchicine is ineffective or not tolerated).
Imbruvica® (ibrutinib) Janssen‐Cilag	Antineoplastic agents (protein kinase inhibitors)	As a single agent for the treatment of adult patients with previously untreated chronic lymphocytic leukaemia.	Yes	Conditionally innovative
Kisquali® (ribociclib) Novartis	Antineoplastic agents	Treatment of women with HR‐positive, HER2‐negative locally advanced or metastatic breast cancer in combination with an aromatase inhibitor as initial endocrine‐based therapy.	No	Conditionally innovative
Maviret® (glecaprevir/pibrentasvir) Abbvie	Antivirals for systemic use	Treatment of chronic hepatitis C virus infection in adults	No	Fully innovative
Ocaliva® (obeticholic acid) Intercept Pharma	Bile and liver therapy	Treatment of primary biliary cholangitis (also known as primary biliary cirrhosis) in combination with ursodeoxycholic acid (UDCA) in adults with an inadequate response to UDCA or as monotherapy in adults unable to tolerate UDCA.	Yes	Non‐innovative
Ocrevus® (ocrelizumab) Roche	Immunosupressants	Treatment of adult patients with early primary progressive multiple sclerosis in terms of disease duration and level of disability, and with imaging features characteristic of inflammatory activity.	No	Non‐innovative
Olumiant® (baricitinib) Eli Lilly	Immunosupressants	Treatment of moderate to severe active rheumatoid arthritis in adult patients who have responded inadequately to, or who are intolerant to one or more disease modifying anti‐rheumatic drugs. Olumiant may be used as monotherapy or in combination with methotrexate.	No	Non‐innovative
Opdivo® (nivolumab) Bristol Myers Squibb	Antineoplastic agents	As monotherapy for the treatment of recurrent or metastatic squamous cell cancer of the head and neck in adults progressing on or after platinum‐based therapy.	No	Conditionally innovative
		As monotherapy is indicated for the treatment of adult patients with relapsed or refractory classical Hodgkin lymphoma after autologous stem cell transplant (ASCT) and treatment with brentuximab vedotin.	No	Conditionally innovative
		As monotherapy for the treatment of locally advanced unresectable or metastatic urothelial carcinoma in adults after failure of prior platinum‐containing therapy.	No	Non‐innovative
Oxervate® (cenegermin) Dompè Farmaceutici	Ophthalmologicals	Treatment of moderate (persistent epithelial defect) or severe (corneal ulcer) neurotrophic keratitis in adults.	Yes	Fully innovative
Prevymis® (letermovir) Merck Sharp Dohme	Antivirals for systemic use	Prophylaxis of cytomegalovirus (CMV) reactivation and disease in adult cytomegalovirus‐seropositive recipients [R+] of an allogeneic haematopoietic stem cell transplant (HSCT).	Yes	Fully innovative
Qarziba® (dinutuximab beta) Eusa Pharma	Antineoplastic agents	Treatment of high‐risk neuroblastoma in patients aged 12 months and above, who have previously received induction chemotherapy and achieved at least a partial response, followed by myeloablative therapy and stem cell transplantation.	Yes	Fully innovative
		In patients with history of relapsed or refractory neuroblastoma, with or without residual disease. Prior to the treatment of relapsed neuroblastoma, any actively progressing disease should be stabilized by other suitable measures.	Yes	Non‐innovative
Revlimid® (lenalidomide) Celgene	Immunosupressants	As monotherapy for the maintenance treatment of adult patients with newly diagnosed multiple myeloma who have undergone autologous stem cell transplantation.	Yes	Conditionally innovative
Rydapt® (midostaurin) Novartis	Antineoplastic agents	In combination with standard daunorubicin and cytarabine induction and high‐dose cytarabine consolidation chemotherapy, and for patients in complete response followed by Rydapt single agent maintenance therapy, for adult patients with newly diagnosed acute myeloid leukaemia who are FLT3 mutation‐positive.	Yes	Fully innovative
		(The assessment of innovativeness refers to the following reimbursed indication only: in combination with standard daunorubicin and cytarabine induction and high‐dose cytarabine consolidation chemotherapy for adult patients with newly diagnosed acute myeloid leukaemia acute myeloid leukaemia who are FLT3 mutation‐positive)
		As monotherapy for the treatment of adult patients with aggressive systemic mastocytosis, systemic mastocytosis (ASM) with associated haematological neoplasm (SM‐AHN), or mast cell leukaemia (MCL).	Yes	Non‐innovative
Spinraza® (nusinersen) Biogen Idec Ltd	Other nervous system drugs	Treatment of 5q spinal muscular atrophy.	Yes	Fully innovative
Stivarga (regorafenib) Bayer	Antineoplastic agents	As monotherapy for the treatment of adult patients with hepatocellular carcinoma (HCC) who have been previously treated with sorafenib.	No	Conditionally innovative
Tecentriq® (atezolizumab) Roche	Antineoplastic agents	As monotherapy for the treatment of adult patients with locally advanced or metastatic NSCLC after prior chemotherapy.	No	Fully innovative (follower)
Vosevi® (sofosbuvir/velpatasvir/voxilaprevir) Gilead	Antivirals for systemic use	Treatment of chronic hepatitis C virus infection in adults	No	Fully innovative
Xermelo® (telotristat etiprate) Ipsen	Other alimentary tract and metabolism products	Treatment of carcinoid syndrome diarrhoea in combination with somatostatin analogue therapy in adults inadequately controlled by somatostatin analogue therapy	Yes	Non‐innovative
Zalmoxis® (allogeneic T cells genetically modified) Molmed	Antineoplastic agents	As adjunctive treatment in haploidentical haematopoietic stem cell transplantation (HSCT) of adult patients with high‐risk haematological malignancies.	Yes	Non‐innovative
Zavicefta® (ceftazidime/avibactam) Pfizer	Antibacterials for systemic use	Treatment of the following infections in adults:	No	Non‐innovative
		‐ Complicated intra‐abdominal infection (cIAI)
		‐Complicated urinary tract infection (cUTI), including pyelonephritis
		‐Hospital‐acquired pneumonia, including ventilator‐associated pneumonia (VAP)
		‐Treatment of infections due to aerobic gram‐negative organisms in adult patients with limited treatment options.
Zejula® (niraparib) Tesaro	Antineoplastic agents	As monotherapy for the maintenance treatment of adult patients with platinum sensitive relapsed high grade serous epithelial ovarian, fallopian tube, or primary peritoneal cancer who are in response (complete or partial) to platinum‐based chemotherapy.	Yes	Conditionally innovative
Zinplava® (bezlotoxumab) Merck Sharp & Dohme	Immune sera and immunoglobulins	Prevention of recurrence of Clostridium difficile infection in adults at high risk for recurrence of C. difficile infection.	No	Non‐innovative