> Drug Development Strategy

FDA allows marketing of new device to help treat carbon monoxide poisoning

The U.S. Food and Drug Administration today allowed marketing of a new device, ClearMate, intended to be used in an emergency room setting to help treat patients suffering from carbon monoxide poisoning. The device uses a novel method for quickly removing carbon monoxide from the body by increasing a patient’s rate of breathing. “Carbon monoxide [...]

2019-03-17T18:28:05+00:0017.03.2019|Drug Development Strategy, Regulatory Affairs|

FDA approves new indication for valve repair device to treat certain heart failure patients with mitral regurgitation

The U.S. Food and Drug Administration today approved a new indication for a heart valve repair device that is intended to reduce moderate-to-severe or severe mitral regurgitation, a leakage of blood backward through the mitral valve into the heart’s left atrium that can cause heart failure symptoms such as shortness of breath, fatigue and swelling [...]

2019-03-17T18:22:25+00:0017.03.2019|Drug Development Strategy, Regulatory Affairs|

FDA approves a new generic valsartan

Today, the U.S. Food and Drug Administration approved a new generic of Diovan (valsartan). Valsartan is an angiotensin II receptor blocker (ARB) that treats high blood pressure and heart failure. The FDA prioritized the review of this drug application to help relieve the recent shortage of this critical medicine as a result of multiple recalls of generic [...]

2019-03-14T22:15:54+00:0014.03.2019|Drug Development Strategy, Regulatory Affairs|

FDA approves new nasal spray medication for treatment-resistant depression; available only at a certified doctor’s office or clinic

The U.S. Food and Drug Administration today approved Spravato (esketamine) nasal spray, in conjunction with an oral antidepressant, for the treatment of depression in adults who have tried other antidepressant medicines but have not benefited from them (treatment-resistant depression). Because of the risk of serious adverse outcomes resulting from sedation and dissociation caused by Spravato [...]

2019-03-10T10:30:36+00:0011.03.2019|Drug Development Strategy, Regulatory Affairs|

ANSM: Pour un accès rapide à l’innovation thérapeutique, toutes les demandes d’ATU nominatives peuvent être dématérialisées via l’application e-Saturne

A partir d’aujourd’hui, toutes les demandes d’Autorisation Temporaire d’Utilisation nominatives (ATUn) de médicaments, peuvent être transmises par les professionnels de santé à l’ANSM via l’application e-Saturne. Celle-ci simplifie les échanges entre l’hôpital et l’Agence afin de donner un accès plus rapide aux traitements en ATUn pour les patients qui en ont besoin. Ce mode de [...]

2019-03-10T10:20:18+00:0010.03.2019|Drug Development Strategy, Regulatory Affairs|

EMA: New add-on treatment to insulin for treatment of certain patients with type 1 diabetes

EMA’s human medicines committee (CHMP) has adopted a positive opinion for Zynquista (sotagliflozin) intended as an adjunct to insulin for certain patients with type 1 diabetes mellitus. Zynquista is a small molecule with dual inhibitor activity on SGLT1 and SGLT2. It works in the kidneys to prevent reabsorption of glucose from the urine and in [...]

2019-03-07T21:30:50+00:0007.03.2019|Drug Development Strategy, Regulatory Affairs|

EMA: First antidote for reversal of anticoagulation with factor Xa inhibitors apixaban and rivaroxaban

EMA’s human medicines committee (CHMP) has recommended granting a conditional marketing authorisation in the European Union for Ondexxya (andexanet alfa). This medicine is to be used as an antidote for adult patients taking the anticoagulant (clot-preventing) medicines apixaban or rivaroxaban, when reversal of their action is needed due to life-threatening or uncontrolled bleeding. Anticoagulants are used to [...]

2019-03-07T21:23:04+00:0007.03.2019|Drug Development Strategy, Regulatory Affairs|

EMA: CHMP recommends authorisation of new treatment for phenylketonuria, a rare inherited metabolic disease

EMA’s human medicines committee (CHMP) has recommended the authorisation of Palynziq (pegvaliase), a new medicine for patients aged 16 and older with phenylketonuria, a rare but potentially serious inherited metabolic disease. Patients suffering from this disorder do not have the enzyme that breaks down phenylalanine, an amino acid which is found in most foods containing [...]

2019-03-04T22:52:15+00:0005.03.2019|Drug Development Strategy, Regulatory Affairs|

EMA: First treatment for rare disease characterised by high levels of triglycerides in blood

The European Medicines Agency has recommended granting a conditional marketing authorisation (CMA) for Waylivra (volanesorsen), the first medicine for the treatment of the familial chylomicronaemia syndrome (FCS). FCS is a rare genetic disease that prevents the body from breaking down fats (lipids). Patients with this condition have extremely high levels of triglycerides in their blood. This causes [...]

2019-03-04T22:49:08+00:0005.03.2019|Drug Development Strategy, Regulatory Affairs|

EMA: New add-on treatment for patients with severe asthma

EMA’s human medicines committee (CHMP) has recommended granting an extension of indication to Dupixent (dupilumab) as an add-on maintenance treatment for adult and adolescent (12 years and older) patients with certain forms of severe asthma. Asthma is a chronic lung disease caused by the interaction of genetic and environmental factors. It causes airways to narrow and swell [...]

2019-03-04T22:35:39+00:0004.03.2019|Drug Development Strategy, Regulatory Affairs|

L’ANSM élargit le périmètre du dispositif accéléré d’autorisation d’essais cliniques (Fast Track) aux essais à design complexe et aux Médicament de Thérapie Innovante (MTI)

Afin de permettre un accès plus rapide aux traitements innovants pour les patients, l’ANSM élargit ses deux circuits courts (Fast Track) d’instruction des demandes d’autorisation d’essais cliniques de médicaments dans le respect de la sécurité des patients. Depuis le 18 février 2019, les essais médicaments à design complexes et les médicaments de thérapie innovante (MTI) [...]

2019-02-21T20:13:43+00:0021.02.2019|Drug Development Strategy, Regulatory Affairs|

EMA: Role of big data for evaluation and supervision of medicines in the EU

Recommendations for a path towards understanding the acceptability of evidence derived from ‘Big data’ in support of the evaluation and supervision of medicines by regulators were published today as part of a summary report of the Heads of Medicines Agencies (HMA) - EMA Joint Big Data task force . The recommendations and associated actions set out what needs [...]

2019-02-19T20:33:22+00:0019.02.2019|Drug Development Strategy, Pharmacovigilance|

FDA authorizes first interoperable insulin pump intended to allow patients to customize treatment through their individual diabetes management devices

The U.S. Food and Drug Administration today permitted marketing of the Tandem Diabetes Care t:Slim X2 insulin pump with interoperable technology (interoperable t:Slim X2) for delivering insulin under the skin for children and adults with diabetes. This new type of insulin pump, referred to as an alternate controller enabled (ACE) infusion pump, or ACE insulin [...]

2019-02-17T20:27:15+00:0018.02.2019|Drug Development Strategy, Regulatory Affairs|

ANSM: Phagothérapie – L’ANSM annonce la création d’un comité scientifique spécialisé temporaire (CSST) intitulé “Phagothérapie – Retour d’expérience et perspectives”

Dans les suites des conclusions du Comité Scientifique Spécialisé Temporaire (CSST) datant de mars 2016, l’Agence nationale de sécurité des médicaments et des produits de santé (ANSM) crée un nouveau comité d’experts nommé « Phagothérapie – Retour d’expérience et perspectives » dont la séance de travail aura lieu le 21 mars prochain. Afin d’échanger sur [...]

2019-02-13T22:18:51+00:0014.02.2019|Drug Development Strategy|

ANSM: Avis et recommandations du Comité des médicaments pédiatriques (PDCO) de l’Agence européenne des médicaments (EMA) – Réunions du 11-14 décembre 2018

Ce Comité est chargé notamment de l’évaluation des PIPs (Plans d’Investigations Pédiatriques) qui encadrent les programmes de développement en qualité, préclinique et clinique des médicaments destinés à la population pédiatrique (de la naissance à 17 ans inclus), ainsi que des dérogations et reports de développement en pédiatrie. Le PDCO vérifie la réalisation du programme d’études [...]

EMA launches checklist to facilitate validation for initial marketing authorisation applications

A survey launched by EMA in September 2016 shows that validation issues occur in 90% of initial marketing authorisation applications (MAAs). They create additional workload for companies and potential delays at a critical moment for the timely start of the procedure. To make the validation process more efficient, predictable and easier to navigate, EMA is encouraging applicants [...]

FDA approves first therapy for the treatment of adult patients with a rare blood clotting disorder

The U.S. Food and Drug Administration today approved Cablivi (caplacizumab-yhdp) injection, the first therapy specifically indicated, in combination with plasma exchange and immunosuppressive therapy, for the treatment of adult patients with acquired thrombotic thrombocytopenic purpura (aTTP), a rare and life-threatening disorder that causes blood clotting. “Patients with aTTP endure hours of treatment with daily plasma [...]

2019-02-10T10:46:46+00:0010.02.2019|Drug Development Strategy, Regulatory Affairs|

Infarmed: Nova área sobre canábis para fins medicinais

No site do Infarmed pode agora encontrar uma área dedicada à canábis para fins medicinais. No seguimento da publicação da Lei n.º 33/2018, de 18 de julho, que estabeleceu o quadro legal para a utilização de medicamentos, preparações e substâncias à base da planta da canábis para fins medicinais - nomeadamente a sua prescrição e a sua dispensa [...]

2019-02-06T21:22:56+00:0007.02.2019|Drug Development Strategy, Regulatory Affairs|

EMA: Meeting highlights from the Committee for Medicinal Products for Human Use (CHMP) 28-31 January 2019

EMA’s human medicines committee (CHMP) recommended six medicines for approval at its January 2019 meeting. The CHMP adopted a positive opinion for Ajovy (fremanezumab), for the prophylaxis of migraine. Vizimpro (dacomitinib) received a positive opinion for the treatment of locally advanced or metastatic non-small cell lung cancer with epidermal growth factor receptor activating mutations. Two biosimilar medicines for the treatment of certain [...]

EMA: First oral add-on treatment to insulin for treatment of certain patients with type 1 diabetes

EMA’s human medicines committee (CHMP) has recommended for the first time an adjunct treatment to insulin in the form of a tablet for certain patients with type 1 diabetes mellitus. Dapagliflozin is already authorised in the European Union as Forxiga and Edistride for the treatment of patients with type 2 diabetes. It belongs to a new generation [...]

2019-02-06T16:16:36+00:0006.02.2019|Drug Development Strategy, Regulatory Affairs|