EMERGING TRENDS IN THE BIOTECH APIs PIPELINE

Aprile 2015

CPA (Italian Chemical Pharmaceutical Association) has recently published the report: Emerging trends in the biotech APIs pipeline After an overview on the global market for the more traditional biotech APIs (including recombinant � proteins, monoclonal antibodies, preventive vaccines and therapeutic enzymes), with a breakdown by product type and by geographical area, as well as on the market for biosimilar APIs, that is predicted to rise at an average 20% yearly over the next few years on a global basis, the report�s core focuses on a detailed analysis on the innovation in the biotech APIs pipeline and its market. The following products are considered in the report: ? advanced therapeutic medicinal products (ATMPs), as defined by the Regulation EC 1394 / 2007: cell therapies, gene therapies, and tissue engineered products; ? RNAi therapies; ? therapeutic vaccines; ? drugs for compassionate use; ? antibody drug conjugates (ADCs). In addition, though many orphan drugs cannot be considered necessarily as �new drugs�, market opportunities for these drugs are also outlined in the report, since they are a strong driver in the biotech APIs market (most orphan drugs are biological products). The number of projects with Orphan Drug Designation has been drastically increasing in the recent past years; of the new drugs approved by the US FDA in the year 2013, nearly 36% were orphan drugs: from the passage of the Orphan Drug Act (ODA) in 1983 until 2013, around 350 � 400 orphan drugs got FDA approval. Though innovative biotech APIs still account for a �niche market� within the global biotech APIs market, they are projected to increase at an average 32 � 33% yearly over the next four years � with RNAi therapies growing even faster than average � as against an average 10% yearly growth forecast for the traditional recombinant proteins� and monoclonal antibodies� market. After a period of �silence� from 2002 to 2008, starting from 2009 a series of stem cell � based products (more than ten) as well as other cell therapies non based on stem cells have got approval; and numerous (stem) cell trials are in the late clinical trial phase. The total number of stem cell clinical trials (sponsored by both industry and Academia) was more than 5,000 as of year 2013; over 40% of these was in the cancer segment. Gene therapies products are drugs that perform a therapeutic effect through modification of the genetic patrimony. Currently two gene therapy products are marketed in China and Philippines respectively, whereas in Europe until now only a gene therapy got approval. A number of companies worldwide are now working for developing gene therapy products, whose main target remains the oncology area, that alone accounts for over 50% of all projects currently under development. Tissue engineered products are engineered cells or tissues having the function of regenerating damaged tissues. Until now two tissue engineering products , both based on chondrocytes, have got regular approval. RNA interference, the biological process in which RNA molecules inhibit gene expression, are opening up interesting opportunities to target RNA. Whereas until two years ago the pharmaceutical giants had disregarded the RNAi area, starting from 2013 the favorable potential of such therapies again attracted the attention of the big players. Though until now only a handful of RNAi therapies have got approval by the US FDA, several RNAi therapies are currently under development. Therapeutic vaccines, unlike traditional vaccines, combat existing diseases rather than offering lasting protection against infections. An example of therapeutic vaccine that can be considered as an �advanced therapy� is Plasmid DNA, whose market is predicted to show abnormal growth in the next few years. Once again, the oncology area is getting most attention by therapeutic vaccines, with two (preventive) cancer vaccines approved by the US FDA, but the R&D activity is engaged also in other therapeutic areas, such as Alzheimer�s disease, infectious disease, diabetes, etc. Several therapeutic vaccines are currently under development. Drugs for compassionate use are not yet approved drugs, still in the clinical trial stage, used to treat seriously ill patients in case no other treatments are available, and when the patient has exhausted all other treatment options. Regulations on drugs for compassionate use vary from one country to another, but in both the US and Europe compassionate use is allowed for drugs under stage III trials. ADCs are new high potent medicines mostly targeted to treat cancer; on the global ADCs� market, North America by far accounts for the majority (about 62%). Some tens of ADCs are currently under development, with a few already approved. The report also analyses the structure of the global biopharmaceutical industry by manufacturing companies� size and by geographical area. The multinational giants (with turnover over $ 500 million) account for around the two thirds of total world revenues. The average turnover of the US biopharmaceutical companies is around 90% higher than average turnover of European companies: in other terms, most biopharmaceutical giants are US companies. Not only, but North America still dominates the global biotech APIs market, accounting for over 40% of world revenues, and for over 55% of the global biotech innovative � emerging therapies market.