Here is an overview of each medicine to help guide you and your physician when making a treatment decision for Cystic Fibrosis: 

Kyowa Hakko Kirin's Parkinson's therapy, Nourianz (istradefylline), gains FDA approval as an add-on to levodopa/carbidopa.

Radicut/Radicava (edaravone), shown to slow the progression of ALS, is now approved for patients in China.

Orkambi and Symkevi have been rejected by the Scotland Medicines Consortium (SMC) due to their conclusion that the costs outweigh the benefits.

Vitrakvi (larotrectinib) recommended by the CHMP for approval in the EU.

New skin cancer medicine now approved in the EU for adults with metastatic or locally advanced cutaneous squamous cell carcinoma.

Vyndaqel (tafamidis) is now approved in the U.S. after study showed increased survivorship rate and reduced hospital time due to heart related problems.

Approval for next phase of clinical trials puts ALS patients one step closer to accessing new treatment shown to slow disease progression.

Based on promising results from phase III clinical trials, Health Canada has decided to extend the use of Kalydeco (ivacaftor) to include children aged 1 to 2 years. 

A recent clinical trial indicates that Symkevi/Symdeko (tezacaftor/ivacaftor) can safely and effectively loosen thick, sticky mucus for children 6-11 years old.

Phase III trial shows that Copiktra (duvelisib) may be an option for patients with relapsed or refractory chronic lymphocytic leukaemia and small lymphocytic lymphoma.

Onpattro (patisiran) is a first-in-class treatment that slows down the progression of hereditary transthyretin amyloidosis (hATTR).

New medicine shows statistically significant improvements for prostate cancer patients.

Australia provides reimbursement for cystic fibrosis medicine.

The European Commission has approved Alunbrig (brigatinib) for the treatment of a specific type of lung cancer.

According to study investigators, about a third of patients could benefit from this therapy that may offer remissions, relieve symptoms and save lives.

2018 has been a good year for new cancer treatments says the U.S. AACR. 
 

Kisqali is now the only CDK4/6 inhibitor for use with an aromatase inhibitor for the treatment of pre-, peri- or postmenopausal women in the US.

The approval marks a significant advance in the field of neurology — and one that migraine patients will no doubt welcome.

The new preventive medicine for migraine Aimovig (erenumab) has been approved by the EMA. Read more...
 

The latest approval renders the previous 'temporary approval' a full approval, without further results needed.

The FDA has approved a wearable device to treat withdrawal symptoms such as agitation, anxiety, depression, and opiate cravings.

Tagrisso (osimertinib) has been approved in Europe to treat Epidermal Growth Factor Receptor (EGFR) mutated non-small cell lung cancer (NSCLC) as a first-line treatment. 

This will provide a much-needed alternative therapy for a debilitating disease with limited treatment options. 



Moderately to severely active ulcerative colitis can now be treated with Xeljanz (tofacitinib), following the expanded approval by the Food and Drug Administration (FDA). This will provide a much-needed alternative therapy for a debilitating disease with limited treatment options.

This new development may increase chances of patients discussing the disease with their doctor...



A new package insert that includes information/data about psoriasis in the genital area has been approved by the Food and Drug Administration (FDA). 

‘Unapproved medicines’, ‘compassionate use’, ‘early access drugs’..