All About Pharmaceuticals

Here’s an example   of the current regulatory framework – you may like it, you may not, but if you’re doing drug research you should know that it’s going on. The way it’s traditionally worked – for decades – has been that a company develops a drug, runs clinical trials, etc., puts together a (huge) data package and sends it off to the FDA. In the oldish days it all went off in hard copy – I remember a celebration when the 18-wheeler pulled out of the loading dock on its way to Maryland, loaded down with box after box of bound copies of the NDA and the associated data. If a company wanted approval for another indication, well, it did something similar: it went out and ran more trials, put a package together, and sent it to the FDA for evaluation. Now, for a long time companies have been working with the agency to try to make this whole process go smoother. You would be well advised to consult with them about whether the trials you’re planning will generate (in their view) dat...

Let’s have a look at the recent new drug approvals. 2018 was quite a year, by the numbers.  C&E News  has  a comprehensive roundup : 59 approvals (versus 46 in 2017, which was already a record by itself), and about two-thirds of those small molecules. There are some very  interesting molecules  in the list, and I always recommend that medicinal chemists sit down every so often and look over the structures of approved drugs as if you’re seeing them for the first time (say, as screening hits). You might be surprised at how many of them you find chemically somewhat unappealing – would you aim for an n-hexyl ether in your final structure ( Mulpleta/lusutrombopag ), the heterocyclic ring in the lower section of  Xofluza (baloxivir marboxil) , or think that  3,4-diaminopyridine (Firdapse)  or  Diacomet (stiripentol)  could be drugs at all? Those last two also illustrate the tricky nature of drug approval statistics. Diaminopyridine ...

💠Stability study ICH guidelines 💢 stability zones conditions in details for long term, intermediate & accelerated stability testing 💨3.2.P.8.2 Post-approval Stability Protocol & Stability Commitment ⚡ A written commitment (signed & dated) is submitted in the following cases: Results of stability studies on three production batches are not completed for all the intervals till the proposed shelf-life period. ⚡ Data from stability studies on less than three production batches. ⚡ Stability data are not on production batches. 💨3.2.P.8.3 Stability Data ⚡Results of the stability studies should be presented in a tabular format. The results of all testing parameters related to each batch for the entire testing period should be presented in one table (i.e. presenting the results of one parameter of all batches in one table is not acceptable). GMP pharmaceutical stability studies and ICH storage services supporting your drug product...

Istradefylline (Nourianz, Kyowa Kirin) tablets have been approved by the US Food and Drug Administration (FDA) as add-on treatment to levodopa/carbidopa in adults who have Parkinson’s disease (PD) experiencing “off” episodes.  Eric Bastings, M.D., acting director of the Division of Neurology Products in the FDA’s Centre for Drug Evaluation and Research said “Parkinson’s disease is a debilitating condition that profoundly impacts patients’ lives.” Istradefylline is a selective adenosine A2A receptor antagonist. When treated on ‘off’ episodes in patients with PD taking levodopa/carbidopa its effectiveness was shown in four 12-week, placebo-controlled clinical studies that included a total of 1143 participants. According to all the four studies, it reports that the addition of istradefylline leads to a statistically significant decrease from baseline in daily “off” time relative to placebo add-on, the FDA stated in a news release. Dyskinesia, dizziness, constipa...

This section provides information on some of the major concepts within pharmacovigilance related to pharmaceutical products for human use . Pharmacovigilance has been defined by the World Health Organisation as “The science and activities relating to the detection, assessment, understanding and prevention of adverse effects or any other possible drug-related problem” Adverse Events & Adverse Reactions The ICH E2A guideline describes Adverse Events as any “untoward medical occurrence” which happens to either a patient or a subject in a clinical investigation when a pharmaceutical product has been given to that person 1 . This encompasses any signs which are unfavourable and unexpected for the patient or subject, including any abnormal laboratory findings. These could be symptoms or a diseases temporally associated with the use of a medicinal product, and do not have to have been previously associated with that product. Neither do they have to have a known causal relations...