Top Regulatory News Stories – Week Ending September 22, 2017

This week the FDA approved two new molecular entities and the EC approved four new programs. Several new orphan drug designations were granted along with a fast track designation.  Here are the top regulatory news stories this week:

Bayer received FDA Approval for Aliqopa™ (copanlisib) 60 mg vial for Injection in adults with Relapsed Follicular Lymphoma after two prior systemic therapies. Accelerated approval was granted for this indication based on objective response rate.

Symbiomix Therapeutics announced that the FDA has approved Solosec™ (secnidazole) 2g oral granules for the treatment of bacterial vaginosis (BV) in adult women.

GSK and Innoviva have secured approval from the FDA for once-daily and single inhaler triple therapy, Trelegy Ellipta, to treat patients with COPD.

Ipsen announced that the European Commission (EC) has approved Xermelo (telotristat ethyl) 250 mg three times a day (tid) for the treatment of carcinoid syndrome diarrhea in combination with somatostatin analogue (SSA) therapy in adults inadequately controlled by SSA therapy.

Novartis’ cancer drug Rydapt (midostaurin) has been approved by the European Commission (EC) for a mutated form of acute myeloid leukemia (AML) and certain rare blood disorders.

Roche has secured approval from the European Commission (EC) for its Gazyvaro (obinutuzumab) in combination with chemotherapy for the treatment for previously untreated advanced follicular lymphoma.

Merck and Pfizer have secured the approval of their skin cancer drug Bavencio (avelumab) from the European Commission (EC) as a monotherapy for the treatment of metastatic Merkel cell carcinoma (mMCC) in adults.

The FDA granted Fast Track Designation for Amicus Therapeutics’ Migalastat for treatment of Fabry Disease. This comes less than a year after the FDA forced Amicus to mount a special trial to collect GI data.

The FDA has granted Motif Bio’s investigational drug candidate, iclaprim, orphan drug designation to treat of Staphylococcus aureus lung infections in patients with cystic fibrosis.

Amylyx Pharmaceuticals announced that the FDA has granted orphan drug designation to AMX0035, an oral therapeutic in clinical development for the treatment of amyotrophic lateral sclerosis (ALS).

ProQR received orphan drug designation from FDA for their drug candidate QR-313 for Dystrophic Epidermolysis Bullosa

PhaseRx, Inc. announced that its second drug development candidate, PRX-ASL, for the treatment of argininosuccinate lyase deficiency (ASLD), has received orphan drug designation by the FDA.

Sunovion submitted an NDA to the FDA for Dasotraline for the treatment of patients with ADHD

Boehringer Ingelheim announced that the Committee for Medicinal Products for Human Use (CHMP) of EMA has adopted a positive opinion for the Marketing Authorization Application of Cyltezo (adalimumab biosimilar), to treat multiple chronic inflammatory diseases in adults and children.

The CHMP also recommended GSK and Innoviva’s fluticasone furoate/ umeclidinium/vilanterol (FF/UMEC/VI) in treating chronic obstructive pulmonary disease (COPD) in adults.

CSL Behring announced that the FDA has approved Privigen®[Immune Globulin Intravenous (Human), 10% Liquid] for the treatment of adults with chronic inflammatory demyelinating polyneuropathy (CIDP) to improve neuromuscular disability. Privigen was initially approved in 2007 for treatment of Primary Immunodeficiency Syndrome.

Janet Woodcock, Director of FDA’s Center for Drug Evaluation and Research, said that the clinical trials system is “broken” and there needs to be new ways to collect and utilize patient data:

In other news…

Ultragenyx proposes to acquire Dimension Therapeutics.  This comes two weeks after the announced acquisition of Dimension by RegenxBio:  

 Alnylam Pharmaceuticals said Wednesday a Phase 3 study testing its experimental therapeutic patisiran met all of its primary and secondary objectives in a rare and debilitating genetic disease:

Pfizer is suing J&J over contracts blocking Remicade biosimilars:

Spark Therapeutics announced it has entered into a licensing agreement with Genethon, a non-profit research and development organization, for the development and commercialization of an adeno-associated viral (AAV) gene therapy targeting the liver to address a rare genetic disease.


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