Compassionate use programma's die zijn goedgekeurd door het CBG Laatst bijgewerkt op: 21 juni 2018

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1 Compassionate use programma's die zijn goedgekeurd door het CBG Laatst bijgewerkt op: 21 juni 2018 Het CBG kan in schrijnende gevallen toestemming verlenen voor het in de handel brengen van een geneesmiddel waarvoor (nog) niet een handelsvergunning is verleend. Dit noemen we het compassionate use programma. Een fabrikant kan deze geneesmiddelen voor de patiëntengroep (cohort) beschikbaar stellen. In dit overzicht staan alle goedgekeurde compassionate use programma s vermeld, inclusief de indicatie en looptijd. Product name (active ingredient) Name of firm Indication Date approval CUP Date of cessation CUP Onpattro (patisiran sodium) Alnylam UK Limited Treatment of adults with stage I-III hattr amyloidosis polyneuropathy (PN) 21 June June 2019 Vyxeos (liposomal formulation of a fixed combination of cytarabin and daunorubicin hydrochloride) Jazz Pharmaceuticals, Ireland Limited. Patients 18 years of age and older with therapy related AML (t-aml) or AML with myelodysplasia-related changes. 18 April April 2019 Imfinzi (durvalumab) Locally advanced, unresectable NSCLC (Stage III), who have not progressed following platinum-based chemoradiation therapy. 21 December December 2018 Alecensa (alectinib hydrochloride) Roche Adult patients with anaplastic lymphoma kinase (ALK)-positive advanced non-small cell lung cancer (NSCLC),and who fulfil the eligibility criteria outlined in the patient profile. January 2017 March 2018

2 Patients with hormone receptor (HR) positive, human epidermal growth factor receptor 2 (HER2) negative locally advanced or metastatic breast cancer: - in combination with an aromatase inhibitor, - in combination with fulvestrant in patients who have received prior endocrine therapy Ibrance (palbociclib) Pfizer B.V. In pre- or perimenopausal women the endocrine therapy should be combined with a luteinizing hormone releasing hormone (LHRH) agonist. 19-jul-16 1 July 2017 Venetoclax Daratumumab (daratumumab) AbbVie B.V. Janssen-Cilag Treatment of adult patients with chronic lymphocytic leukaemia (CLL) in the presence of 17p deletion or TP53 mutations who are insuitable for or have failed a B-cell receptor pathway inhibitor Multiple myeloma - patients who have relapsed and refractory multiple myeloma (MM), who have received at least 3 prior lines of therapy and who are unsuitable for retreatment with a proteasome (PI) and an immunomodulatory agent (IMiD), or whose disease is double refractory to both a PI and an IMiD 21 June June February 2016 Tagrisso (osimertinib) Adult patients with locally advanced or metastatic epidermal growth factor receptor (EGFR) T790M mutation-positive non-small-cell lung cancer (NSCLC) who have progressed on or after EGFR TKI therapy. 14 January May 2016

3 Lonsurf (trifluridine + tipiracil) Servier Nederland Farma BV Adult patients with metastatic colorectal cancer (CRC) who have been previously treated with, or are not considered candidates for, available therapies including fluoropyrimidine-, oxaliplatin- and irinotecan-based chemotherapies, anti-vegf agents, and anti- EGFR agents. 17 December December 2016 Nivolumab BMS (nivolumab) Bristol-Myers Squibb B.V. Patients with histologically- or cytologicallydocumented SqNSCLC and non-sqnsclc who have relapsed after systemic pretreatment with a minimum of 1 prior systemic treatment for stage IIIB/stage IV disease 18 May May 2016 Cotellic (cobimetinib) Roche Nederland B.V. For use in combination with Zelboraf (vemurafenib) for treatment-naïve adult patients with BRAF V600 mutation-positive unresectable or metastatic melanoma 9 March March 2016 Lynparza (olaparib) Monotherapy for the maintenance treatment of adult patients with platinum sensitive relapsed BRCA mutated (germline and/or somatic) high grade serous epithelial ovarian, fallopian tube, or primary peritoneal cancer who are in response (complete response or partial response) to platinum-based chemotherapy 15 December May 2015 Keytruda (pembrolizumab) Merck Sharp & Dome Patients with a diagnosis of unresectable (Stage III) or metastatic melanoma (Stage IV) who have failed or progressed on standard-of-care systemic therapy including ipilimumab, and when indicated a BRAF/MEK inhibitor 03 November November 2015

4 Nintedanib Boehringer Ingelheim Treatment of patients with Idiopathic Pulmonary Fibrosis (IPF) 5 June March 2015 Daklinza (daclatasvir) Bristol-Myers Squibb B.V. Subjects chronically infected with hepatitis C who are at a high risk of liver decompensation or death within 12 months if left untreated and who have no available therapeutic options 14 April March 2015 Imbruvica (ibrutinib) Johnson & Johnson / Janssen-Cilag B.V. Treatment of adult patients with relapsed or refractory: mantle cell lymphoma (MCL), chronic lymphocytic leukemia (CLL) or small lymphocytic lymphoma (SLL) 10 April November 2014 Cyramza (ramucirumab) Dafrafenib (trametinib) Lilly Nederland B.V. GlaxoSmithKline Cyramza in combination with paclitaxel is indicated for the treatment of adult patients with advanced gastric cancer or gastrooesophageal junction adenocarcinoma with disease progression after prior platinum and fluoropyrimidine chemotherapy. Cyramza monotherapy is indicated for the treatment of adult patients with advanced gastric cancer or gastro-oesophageal junction adenocarcinoma with disease progression after prior platinum or fluoropyrimidine chemotherapy, for whom treatment in combination with paclitaxel is not appropriate. Unresectable or metastatic melanoma with BRAF V600 mutation who did not receive previous treatment with BRAF- and/or MEK inhibitors 3 March June June 2014 This medicine has been given a marketing authorisation for the Netherlands, the Compassionate Use Programme has therefore ended as of 5 February 2015.

5 Kadcyla (trastuzumab-emtansine (T-DM1)) Roche Metastasised breast cancer. Have received at least two lines of Herceptin-containing regimens in combination with two different forms of chemotherapy (anthracyclines, taxanes and capecitabine) for the metastasised disease in addition to a minimum of one line of lapatinib 7 January February 2014 Zytiga (abiraterone) Tamiflu i.v. (oseltamvir) Relenza i.v. (zanamivir) Janssen-Cilag Roche Nederland B.V. GlaxoSmithKline Treatment of metastatic castration resistant prostate cancer in adult men whose disease has progressed on or after a docetaxel-based chemotherapy regimen 25 November December 2011 Patients with an influenza infection who are unable to take, tolerate or absorb oral formulations and for whom intravenous administration of an intravenous neuraminidase inhibitor is indicated Treatment of severe influenza in critically ill hospitalised patients 12 October October October June 2014 Jevtana (cabazitaxel) Sanofi-Aventis Treatment of patients with hormone refractory metastatic prostate cancer previously treated with a docetaxel-containing regimen 5 June June 2011